PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC begins rolling NDA submission for Translarna to treat nmDMD: "PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has commenced a rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). A rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis. PTC expects to finalize the application in the fourth quarter of 2015 following the completion of the ACT DMD confirmatory Phase 3 clinical trial."



'via Blog this'

» Sarepta Thereaputics release information on trial for SRP-4053 for skipping exon 53 Action Duchenne

» Sarepta Thereaputics release information on trial for SRP-4053 for skipping exon 53 Action Duchenne: "Sarepta Therapeutics have released information regarding the clinical trial for their drug candidate SRP-4053 for skipping exon 53, which is taking place in Europe.

Enrolment for the London centre at Great Ormond Street Hospital is currently taking place, with the Newcastle Centre planned for the future.
 
Eligibility criteria and other information is included in the posting on the clinical trials website.

 

Inclusion criteria includes:

 

being amenable to exon 53 skipping,
stable pulmonary and cardiac function,
minimum performance on 6 minute walk test, North Star Ambulatory Assessment, and rise (Gowers) test as specified in the study protocol,
On a stable dose of corticosteroids for at least 6 months.
Aged between 6-15 years old
Intact right and left biceps muscles or an alternative upper arm muscle group."



'via Blog this'

» European early access programme for Carmeseal-MD(TM) Action Duchenne

» European early access programme for Carmeseal-MD(TM) Action Duchenne: "Phrixus Pharmaceuticals aims to demonstrate that Carmeseal-MD has beneficial effects in patients with Duchenne and Becker muscular dystrophy by delivering an improvement in cardiac and respiratory function via protection of heart muscle and diaphragm.   The drug acts as a molecular band-aid by binding to, and then sealing, microscopic tears in muscle cells caused by the lack of functional dystrophin.  This prevents the uncontrolled leakage of calcium which in turn increases the performance of heart muscle and diaphragm and prevents their degeneration."



'via Blog this'

» NHS Delays Decision on Translarna Action Duchenne

» NHS Delays Decision on Translarna Action Duchenne: "Action Duchenne has been informed that NHS England has delayed the CPAG meeting that was due to take place next week on the 9th of December. The meeting has been cancelled as NHS England are re-considering how it assesses new treatments.

We are extremely concerned and disappointed about this decision. You will recall that from our conference that the complex and overly long commissioning process for new treatments was called into question by our CEO. This delay of the CPAG is symptomatic of the fragile commissioning process."



'via Blog this'

» Please sign petition for full funding Translarna Action Duchenne

» Please sign petition for full funding Translarna Action Duchenne: "We would like to ask that our members support & sign the petition below for each of the UK NHS authorities to fully fund the costs for Translarna, which would be the first treatment for Duchenne to become available to patients in the UK.

Action Duchenne’s staff have been working for the previous 3 months with NHS England, PTC Therapeutics and other UK Duchenne charities to ensure that those who are in the decision making process have been made fully aware of the importance of funding of this first treatment for Duchenne."



'via Blog this'

» BioMarin to purchase Prosensa and prioritise licensing for drisapersen Action Duchenne

» BioMarin to purchase Prosensa and prioritise licensing for drisapersen Action Duchenne: "BioMarin Pharmaceutical have agreed to purchase Prosensa for $680 million , with two further payments of $80 million subject to drisapersen , Prosensa’s leading candidate for skipping exon 51 in Duchenne, being licensed in the USA by 15th May 2016 and Europe no later than 15th February 2017.

 

Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin said “BioMarin is dedicated to the rare disease community, and the acquisition of Prosensa fits strategically with our mission of delivering therapies that address serious unmet medical needs. We are committed to working closely with regulatory authorities worldwide in bringing a potentially breakthrough therapy to patients with this devastating condition.”"



'via Blog this'

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy: "Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease."



'via Blog this'

» Sarepta Therapeutics Announces First Patient Dosed in Study of Non-Ambulant Patients with DMD Action Duchenne

» Sarepta Therapeutics Announces First Patient Dosed in Study of Non-Ambulant Patients with DMD Action Duchenne: "On the 12th of November, Sarepta announced the initiation for dosing in the 204 clinical study for non-ambulant and advanced DMD patients. The link to the official press release can be found here: http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsArticle&ID=1989115

This study includes around 20 patients treated with eteplirsen whom can particularly be treated with exon 51 skipping. The investigation is being carried out in the United States and the major aims are to evaluate the safety of eteplirsen in DMD patients over a dosing time period of 96 weeks."



'via Blog this'

» ITV NEWS COVERAGE- Are Drug Companies doing enough to tackle Duchenne? Action Duchenne

» ITV NEWS COVERAGE- Are Drug Companies doing enough to tackle Duchenne? Action Duchenne: "On the 17th October, Action Duchenne’s CEO Paul Lenihan was asked to appear on ITV News to provide further background to Carl Tilson’s discussions with Summit plc.

Paul emphasised throughout his interview that Action Duchenne is committed to early and free access to all new Duchenne medicines regardless of funding and cost and that all parties should combine to influence the legislative processes – processes that currently could delay such early access to medicine. We remain committed to this line and hope this article will add to the debate surrounding the Medical Innovation Bill.

To see the interview in full, click here."



'via Blog this'

PPMD Submits Letter to FDA Regarding Latest Sarepta Update - PPMD Community

PPMD Submits Letter to FDA Regarding Latest Sarepta Update - PPMD Community: "Today’s press release and investor call from Sarepta have sent a ripple of angst and sadness through our community. Moments ago, PPMD sent a letter to the FDA that reflects our organization’s belief that safety and rigor have been demonstrated throughout this process. Further, we believe that any strengthening of data packages that causes delays (as is now being requested by the FDA, as articulated in today’s press release and call) should ensure a streamlined, more expeditious approval process for follow-on exons."



'via Blog this'

» Prosensa begins NDA submission to the FDA for exon-skipping drug drisapersen to treat Duchenne muscular dystrophy Action Duchenne

» Prosensa begins NDA submission to the FDA for exon-skipping drug drisapersen to treat Duchenne muscular dystrophy Action Duchenne: "Prosensa begins NDA submission to the FDA for exon-skipping drug drisapersen to treat Duchenne muscular dystrophy"



'via Blog this'

» Prosensa extends the re-dosing of drisapersen in Europe in patients with Duchenne muscular dystrophy Action Duchenne

» Prosensa extends the re-dosing of drisapersen in Europe in patients with Duchenne muscular dystrophy Action Duchenne: "Prosensa extends the re-dosing of drisapersen in Europe in patients with Duchenne muscular dystrophy"



'via Blog this'

» Prosensa announces Lancet Neurology publication of an exploratory phase II study of Drisapersen Action Duchenne

» Prosensa announces Lancet Neurology publication of an exploratory phase II study of Drisapersen Action Duchenne: "Prosensa announces Lancet Neurology publication of an exploratory phase II study (DEMAND II) demonstrating efficacy and safety of drisapersen in patients with Duchenne muscular dystrophy"



'via Blog this'

» World Duchenne Awareness Day Action Duchenne

» World Duchenne Awareness Day Action Duchenne: "On the 7th of September 2014 the First World Duchenne Awareness Day will take place. The 7th of the 9th has been chosen to represent the 79 exons in the dystrophin gene. Duchenne Charities around the world will raise awareness for boys and young men living with Duchenne muscular dystrophy."



'via Blog this'

» World Duchenne Awareness Day Action Duchenne

» World Duchenne Awareness Day Action Duchenne: "On the 7th of September 2014 the First World Duchenne Awareness Day will take place. The 7th of the 9th has been chosen to represent the 79 exons in the dystrophin gene. Duchenne Charities around the world will raise awareness for boys and young men living with Duchenne muscular dystrophy."



'via Blog this'

» Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy Action Duchenne

» Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy Action Duchenne: "Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy"



'via Blog this'

New gene therapy protects mice from life-threatening heart condition

New gene therapy protects mice from life-threatening heart condition: "A new gene therapy developed by researchers at the University of Missouri School of Medicine has been shown to protect mice from a life-threatening heart condition caused by muscular dystrophy.

"This is a new therapeutic avenue," said Yi Lai, Ph.D., the leading author of the study and assistant research professor in the MU School of Medicine's Department of Molecular Microbiology and Immunology. "This is just a first step, but we hope this could lead to a treatment for people with this devastating heart condition, which is a leading cause of death for people with Duchenne muscular dystrophy.""



'via Blog this'

Researchers use new gene editing method to correct mutation that leads to DMD

Researchers use new gene editing method to correct mutation that leads to DMD: "UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition.

Researchers used a technique called CRISPR/Cas9-mediated genome editing, which can precisely remove a mutation in DNA, allowing the body's DNA repair mechanisms to replace it with a normal copy of the gene. The benefit of this over other gene therapy techniques is that it can permanently correct the "defect" in a gene rather than just transiently adding a "functional" one, said Dr. Eric Olson, Director of the Hamon Center for Regenerative Science and Medicine at UT Southwestern and Chairman of Molecular Biology."



'via Blog this'

» Translarna Press Release -First ever treatment for rare muscle wasting condition, Duchenne Muscular Dystrophy, given conditional approval by the European Commission Action Duchenne

» Translarna Press Release -First ever treatment for rare muscle wasting condition, Duchenne Muscular Dystrophy, given conditional approval by the European Commission Action Duchenne: "Translarna Press Release -First ever treatment for rare muscle wasting condition, Duchenne Muscular Dystrophy, given conditional approval by the European Commission"



'via Blog this'

Splice-switching targeted oligonucleotide drugs alter editing of gene transcript

Splice-switching targeted oligonucleotide drugs alter editing of gene transcript: "In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. Developments in this rapidly advancing field have already led to promising treatments for such diseases as Duchenne Muscular Dystrophy and spinal muscular atrophy, as described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website."



'via Blog this'

Illinois Designates Week of June 23 as Duchenne Awareness Week! - PPMD Community

Illinois Designates Week of June 23 as Duchenne Awareness Week! - PPMD Community: "The State of Illinois is recognizing our 20th Annual Connect Conference in Chicago, Illinois, June 26-29, 2014 by declaring the week of June 23 as Duchenne Muscular Dystrophy Awareness Week! With over 500 families and Duchenne experts expected to attend from around the world, this will be our largest Annual Connect Conference to date."



'via Blog this'

Webinar: Ataluren (Translarna) Update with PTC - PPMD Community

Webinar: Ataluren (Translarna) Update with PTC - PPMD Community: "In a webinar hosted by United Parent Project Muscular Dystrophy, Robert Spiegel, MD, FACP, chief medical officer of PTC Therapeutics, presented an overview of the positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency regarding the company’s application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, and what this decision means for families living in and outside the EU."



'via Blog this'

Summit Corp establishes US office | 17 June 2014 | Stock Market Wire

Summit Corp establishes US office | 17 June 2014 | Stock Market Wire: "Summit Corp has established US operations in Cambridge, Massachusetts and strengthened its drug development operations team following the recruitment of new clinical and preclinical employees.

The US operations are being formed to facilitate greater interactions with academic, clinical and business leaders in Summit's areas of therapeutic development.

Located at the new US office will be key members of the clinical development team: Dr Michael Boss, the overall leader of the utrophin modulation programme for DMD, and Dr Bindu Tejura who recently joined Summit as Vice President of Clinical Development and will be involved in both the DMD and CDI programmes."



'via Blog this'

Clinical Development of HT-100 for Duchenne Has Resumed - PPMD Community

Clinical Development of HT-100 for Duchenne Has Resumed - PPMD Community: "Phase 1b/2a Clinical Program Resumes Enrollment and Dosing in DMD Patients 
Preliminary Clinical Data to be presented at Upcoming Medical Conference "



'via Blog this'

» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne

» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne: "DART Therapeutics announced yesterday that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-releasehalofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. The company will continue to enroll and dose patients in this ongoing phase 1b/2a clinical study to evaluate the safety and tolerability of increasing doses of HT-100, and explore trends in a range of efficacy endpoints.

Please see the link for the press release for more information:"



'via Blog this'

» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne

» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne: "DART Therapeutics announced yesterday that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-releasehalofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. The company will continue to enroll and dose patients in this ongoing phase 1b/2a clinical study to evaluate the safety and tolerability of increasing doses of HT-100, and explore trends in a range of efficacy endpoints.

Please see the link for the press release for more information:"



'via Blog this'

» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne

» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne: "DART Therapeutics announced yesterday that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-releasehalofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. The company will continue to enroll and dose patients in this ongoing phase 1b/2a clinical study to evaluate the safety and tolerability of increasing doses of HT-100, and explore trends in a range of efficacy endpoints.

Please see the link for the press release for more information:"



'via Blog this'

PPMD Benefit/Risk Study Results Published in Clinical Therapeutics - PPMD Community

PPMD Benefit/Risk Study Results Published in Clinical Therapeutics - PPMD Community: "PPMD is thrilled that results from the recent benefit/risk project our team led have been published in Clinical Therapeutics. The article concludes that caregivers are willing to accept a serious risk when balanced with noncurative slowing or stopping of the progression of muscle weakness, even absent improvement in lifespan. These preferences should inform the Food and Drug Administration’s (FDA’s) benefit–risk assessment of emerging Duchenne therapies."



'via Blog this'

Lend Your Voice to the FDA Draft Guidance on Duchenne to Speed the Development of Therapies - PPMD Community

Lend Your Voice to the FDA Draft Guidance on Duchenne to Speed the Development of Therapies - PPMD Community: "For the past four months, over 80 experts in Duchenne (including patient representatives and groups, industry, clinicians, and researchers) have been working hard to produce draft guidance on Duchenne that we believe will ultimately speed the development of therapies. "



'via Blog this'

Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne - PPMD Community

Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne - PPMD Community: "Prosensa Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval"



'via Blog this'

Congressional Committee That Funds the FDA Recognizes PPMD-Led Efforts on Benefit/Risk, Draft Guidance; Applauds Collaboration with Agency - PPMD Community

Congressional Committee That Funds the FDA Recognizes PPMD-Led Efforts on Benefit/Risk, Draft Guidance; Applauds Collaboration with Agency - PPMD Community: "For the first time ever, the appropriations bill that funds the Food and Drug Administration includes report language focused on Duchenne muscular dystrophy and championed by PPMD advocates."



'via Blog this'

Webinar: Ataluren (Translarna) Update with PTC - PPMD Community

Webinar: Ataluren (Translarna) Update with PTC - PPMD Community: "In a webinar hosted by United Parent Project Muscular Dystrophy, Robert Spiegel, MD, FACP, chief medical officer of PTC Therapeutics, will present an overview of the positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency regarding the company’s application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, and what this decision means for families living in and outside the EU."



'via Blog this'

» Prosensa update on regulatory discussions and ongoing plans for re-dosing Action Duchenne

» Prosensa update on regulatory discussions and ongoing plans for re-dosing Action Duchenne: "Prosensa today provided an update to the patient-led organisations. They have been encouraged in their discussions with the regulatory authorities and are on-track for a pathway forward by the end of June.

The team also informed us that they are also on-track for re-dosing in the first group of boys in the third quarter of this year. Re-dosing is taking a staged approach, under new or existing treatment protocols or via expanded access programs. In a first wave, they will initiate re-dosing at sites in both US and Europe and as stated previously are actively working with these sites and investigators to execute these plans."



'via Blog this'

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna: "PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company's application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older."



'via Blog this'

Summit updates on Phase 1b trial for SMTC1100 | 21 May 2014 | Stock Market Wire

Summit updates on Phase 1b trial for SMTC1100 | 21 May 2014 | Stock Market Wire: "StockMarketWire.com - Summit Corporation says SMT C1100 for the treatment of Duchenne Muscular Dystrophy (DMD) has successfully met its primary endpoint of safety and tolerability in a Phase 1b clinical trial in patients with the disease.

SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease.

The Phase 1b dose-escalating trial was conducted in 12 patients with DMD aged between 5 and 11 years old.

"These preliminary results show that SMT C1100 was safe and well tolerated at all doses tested in the study, and that there were no issues with patient compliance," Summit said. "



'via Blog this'

20th Annual Connect Conference - Parent Project Muscular Dystrophy

20th Annual Connect Conference - Parent Project Muscular Dystrophy: "This year marks PPMD's 20th Annual Connect Conference in Chicago, June 26-29, 2014. What started as a few dozen parents and a handful of experts gathered to discuss and learn all they could about Duchenne, has grown into a meeting where families, physicians, researchers, industry leaders, and experts of all kinds gather to speak face-to-face about Duchenne."



'via Blog this'

Unprecedented Collection of Duchenne Experts Developing Guidance for FDA - PPMD Community

Unprecedented Collection of Duchenne Experts Developing Guidance for FDA - PPMD Community: "The process for writing a Draft Guidance on Duchenne for the FDA and industry is in full swing.
As you know, the purpose of this guidance is to assist sponsors in the clinical development of medical products (i.e., human drugs and therapeutic biological products) for the treatment of Duchenne over the entire spectrum of the disease (ambulatory and non ambulatory). It is the result of the first collaboration between the FDA and a rare disease community to produce clinical guidance in their respective disease area."



'via Blog this'

Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne - PPMD Community

Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne - PPMD Community: "A recent PPMD-funded study at Cedars Sinai Medical Center in Los Angeles (published in the May 7, 2014, online issue of Neurology®) demonstrates that those with Duchenne have impaired circulation in their muscles, which may contribute to muscle weakness, and that the approved vasodilator tadalafil can restore that circulation to normal levels. "



'via Blog this'

DuchenneConnect Annual Report: Registry Growth & Expanded Services - PPMD Community

DuchenneConnect Annual Report: Registry Growth & Expanded Services - PPMD Community: "This past year has brought many new and exciting changes to DuchenneConnect, the robust and cutting-edge registry and resource that connects Duchenne and Becker patients with actively recruiting clinical trials and research studies, and educates patients and families about Duchenne and Becker research. The registry, which was established in 2007, has matured beyond a standard registry and into an innovative resource for the entire Duchenne and Becker community. This 2013 Annual Report (PDF) highlights how we have grown and expanded our services over the past year. "



'via Blog this'

» Drisapersen update from Prosensa Action Duchenne

» Drisapersen update from Prosensa Action Duchenne: "Prosensa issued an update today to the parent-led organisations on their lead exon skipping compound to skip exon 51, drisapersen; following their recent UPPMD webinar on the 25th of March.
Whilst Prosensa are finalising the transfer from GSK, they also continue to embark on their program to re-dosing the first cohort of boys who participated in the trial in the third quarter of this year and also communicating their plans and regulatory paths forward forward, by the end of June."



'via Blog this'

Webinar: Sarepta Therapeutics - PPMD Community

Webinar: Sarepta Therapeutics - PPMD Community: "Sarepta Therapeutics, Inc. will host a webcast and conference call for the Duchenne muscular dystrophy (DMD) community on Tuesday, May 13, 2014, beginning at 11:00 a.m. EDT (8:00 a.m. PDT / 5:00 p.m. CEST).

Chris Garabedian, president and chief executive officer, and Edward Kaye, M.D., chief medical officer, will provide information and answer questions about Sarepta's DMD development program, including upcoming clinical trials. Sarepta has invited DMD community representatives from leading patient advocacy groups to participate in the question and answer portion of the webcast."



'via Blog this'

At Long Last, A Victory! - PPMD Community

At Long Last, A Victory! - PPMD Community: "This morning, Sarepta announced that its discussions with the Food and Drug Administration (FDA) over the last several months have borne fruit—the FDA has agreed to allow Sarepta to submit a New Drug Application (NDA) for eteplirsen.  The company plans to submit the application by the end of this year and should have a decision from the FDA by mid-2015 about whether or not it will be approved.   Over the last several years, the chances that a drug will go on to be approved after an NDA is allowed is well over 50%. "



'via Blog this'

Sarepta Therapeutics to Submit New Drug Application to FDA for Eteplirsen by the end of 2014 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Sarepta Therapeutics to Submit New Drug Application to FDA for Eteplirsen by the end of 2014 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Sarepta Therapeutics announced yesterday their plans to submit a new drug application (NDA) for eteplirsen to the US Food and Drug Administration (FDA) by the end of this year. Eteplirsen is Sarepta's lead candidate to skip exon 51."



'via Blog this'

Clinical trial recruitment updates - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Clinical trial recruitment updates - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Action Duchenne are in continuous dialogue with healthcare professionals, leading biotech and pharmaceutical companies regarding their growing Duchenne pipeline. A snapshot of clinical trial recruitment updates are below.

FOR-DMD (Finding the Optimum Regimen of Corticosteroids for DMD)

This compares daily prednisone and deflazacort regimes against one of the more commonly used intermittent regimes in boys living with Duchenne. "



'via Blog this'

Certified Duchenne Care Center Program - PPMD Community

Certified Duchenne Care Center Program - PPMD Community: "Right now, there are more than 10-12,000 young people diagnosed and living in the United States with Duchenne. These young people are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Families want care and treatment to be transparent; they want to know who is delivering care to their child, what comprehensive care looks like, and which centers are delivering that care. For this reason, a group of experts agreed to give families the information that they deserve and to develop a methodology and process by which to certify clinics delivering comprehensive care, and then measure results.

PPMD’s Transforming Duchenne Care Initiative (TDCI) set out to reduce discrepancies in care. The goal – to make comprehensive care, aligned with the recommendations of the DMD Care Considerations Working Group (DMDWG, which published the CDC Care Considerations standards of Duchenne care, made available in 2009 and published in 2010), available and accessible to as many patients across the US as possible."



'via Blog this'

Webinar: Prosensa Update on Drisapersen - PPMD Community

Webinar: Prosensa Update on Drisapersen - PPMD Community: "Prosensa presented a status update on Drisapersen and its follow-on compounds in a patient focused webinar hosted by United Parent Projects Muscular Dystrophy (UPPMD) on Tuesday, March 25th at 1 PM ET.

The webinar was presented by Giles Campion MD, Chief Medical Officer and Senior Vice-­‐President R&D of Prosensa and featured the following guest speakers:
Craig Campbell MD MSc FRCPC, Head of Section of Pediatric Neurology at London Health Sciences Centre, Associate Professor at Shulich School of Medicine of Western University, Associate Scientist at Child Health Research Institute
Craig M. McDonald MD, Professor and Chair of Physical Medicine & Rehabilitation, Professor of Pediatrics at University of California Davis School of Medicine"



'via Blog this'

Parents of boys with Duchenne urge drug OK - Pittsburgh Post-Gazette

Parents of boys with Duchenne urge drug OK - Pittsburgh Post-Gazette: "A Coraopolis boy is one of a dozen patients with Duchenne muscular dystrophy participating in a clinical trial for the drug eteplirsen, of which FDA approval remains elusive."



'via Blog this'

Device to improve blood flow implanted in muscular dystrophy patient

Device to improve blood flow implanted in muscular dystrophy patient: ""Today, we're going to make history," said 18-year-old Eric Ramos on the day UT Southwestern Medical Center doctors operated on his ailing heart. Eric, who has Duchenne muscular dystrophy, is one of only three patients in the United States with the condition to receive a battery-operated left ventricular assist device (LVAD) to keep his weakening heart pumping blood through his body. He is the first patient in the country to be given a specific, smaller LVAD, which means doctors would not need to manipulate his diaphragm, which could compromise his already limited pulmonary function."



'via Blog this'

Empower: Access to Medicine Parliamentary Event brings Duchenne community together - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Empower: Access to Medicine Parliamentary Event brings Duchenne community together - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "Yesterday, Kathy Wedell, the chair of Action Duchenne and Andy Soar, Campaign Officer for Action Duchenne, attended the Empower: Access to Medicines Event at parliament.

Families, patients, researchers, politicians and pharma companies packed the Strangers Dining Room to celebrate the announcement of the UK Early Access scheme and the Adaptive Licensing Pilot in Europe (http://www.actionduchenne.org/viewarticle?news=519).

This scheme is good news for conditions which have potential treatments in the pipeline.
"



'via Blog this'

Action Duchenne International Conference 2014 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne International Conference 2014 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "We are pleased to announce that our International Conference will be held at the Institute of Education on the 7th and 8th of November. Entitled 'From the lab to the clinic - opportunities for the Duchenne community' the conference will again comprise presentations from the international Duchenne research community.

Running parallel to the Conference, Action Duchenne is delighted that Takin' Charge is planning its largest workshop in its exciting and forward-leaning programme. The gala dinner will be held in the Bloomsbury Holiday Inn on Fri 7 November - this is a 5 minute walk from the venue - and we are delighted to announce that the Holiday Inn have sponsored us by offering considerable discounted Early bird booking rates. Double rooms are available at £161.10 until the 31 May and then at £170.05 from 1st June until 31 August. Rooms booked after this time will attract the full rate of £250 per night. "



'via Blog this'

PROSENSA RELEASE 48 WEEK DATA FROM DRISAPERSEN STUDY

Prosensa today released 48-week drisapersen data from its U.S.-based, Phase II placebo-controlled study (DMD114876 or DEMAND V).

The results of this study indicate that, compared to placebo, some boys in the higher-dose drisapersen group (6 mg/kg once weekly) reported improvements in their muscle function and physical activity as measured by the six-minute walk test (6MWT) for the 24-week treatment phase and maintained this improvement during the 24-week follow-up period.

EUROPEAN MEDICINES AGENCY LAUNCHES ADAPTIVE LICENSING PILOT PROJECT

The adaptive licensing approach launched this week by the European Medicines Agency (EMA), aims to improve timely access for patients to new medicines.

This newly implemented process starts with the early authorisation of a medicine in a restricted patient population, followed by iterative phases of evidence gathering and adaptations of the marketing authorisation to expand access to the medicine to broader patient populations.

Building on their existing mechanisms, centralised compassionate use and conditional marketing authorisation; the EMA intends to include as many programmes as necessary in this pilot phase in order to gather sufficient knowledge and experience. Companies are invited to submit proposals for experimental medicines in the early stage of clinical development.

News Release | sareptatherapeutics.com

News Release | sareptatherapeutics.com: Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced positive safety results from a Phase I multiple ascending dose study of AVI-7288 in healthy volunteers. AVI-7288, which uses Sarepta’s advanced and proprietary PMOplus™ chemistry, is the company’s lead drug candidate for the treatment of Marburg virus infection. Sarepta has been developing AVI-7288 under a Department of Defense contract managed by the Medical Countermeasure Systems BioDefense Therapeutics (MCS-BDTX) Joint Product Management Office.