Expanding the Duchenne Therapeutic Opportunities by Targeting Myostatin - PPMD Community

Expanding the Duchenne Therapeutic Opportunities by Targeting Myostatin - PPMD Community: "We all understand that Duchenne muscular dystrophy is characterized by muscle wasting and associated loss of function and that there are considerable efforts underway to develop drugs and biologics (cell and gene therapy) to address the primary problem in Duchenne—the absence of dystrophin. Restoring dystrophin or replacing dystrophin with replacement protein are considered foundational therapies.
 
That said, the Duchenne community is well aware of the need for combination therapies. Combinations would include compounds that target what is referred to as the ‘downstream pathology’ or the changes that occur because dystrophin is absent. This includes anti-inflammatories, anti-fibrotics, factors that control muscle regeneration and fiber size, compounds that improve circulation to muscle, and compounds that improve mitochondrial function (mitochondria are considered the powerhouses of cells). We are all hopeful that by combining several of these targeted therapies, we could end Duchenne, stop progression for every individual. This is the dream of precision or personalized medicine. The right drug, at the right time, in the right dose for the right person. It requires planning and it will require combinations."



'via Blog this'

Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner

Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner: "Statins are widely known for their use in improving cardiovascular health through lowering blood cholesterol levels. What prompted you to study their use in Duchenne muscular dystrophy (DMD)?"



'via Blog this'

» Translarna Update: Action Duchenne submit FOI Requests to HM Treasury and the Department of Health Action Duchenne

» Translarna Update: Action Duchenne submit FOI Requests to HM Treasury and the Department of Health Action Duchenne: "Translarna Update: Action Duchenne submit FOI Requests to HM Treasury and the Department of Health
Posted on: July 20th, 2015 | 1 comment

Action Duchenne have submitted Freedom of Information (FOI) requests to both Her Majesty’s Treasury and the Department of Health as part of the ongoing campaign for access to Translarna."



'via Blog this'

Study finds that muscle fibrosis inhibition causes increases in utrophin levels and revertant myofibers in Duchenne Muscular Dystrophy

Study finds that muscle fibrosis inhibition causes increases in utrophin levels and revertant myofibers in Duchenne Muscular Dystrophy: "In a recent study published in Oncotarget, researchers found an inverse correlation between the level of muscle fibrosis, utrophin and the number of revertant myofibers in Duchenne Muscular Dystrophy (DMD).

Results from this study reveal common links between the fibrotic and utrophin-synthesis pathways and offer new insights into the regulation of utrophin synthesis in Duchenne Muscular Dystrophy."



'via Blog this'

» PTC Therapeutics fundraising endeavours for Action Duchenne Action Duchenne

» PTC Therapeutics fundraising endeavours for Action Duchenne Action Duchenne: "PTC Therapeutics are sponsoring the LEJOG 2015 Challenge, which takes place between 26th July and 4 August.

Action Duchenne has 21 riders taking part in LEJOG 2015, including three relay teams. If you would like to support the challenge in fundraising or get involved in a similar event please contact Paul Bown via 020 8556 9955 or paulbown@actionduchenne.org

Lands End to John O’Groats is one of the most iconic cycle tours around , taking in some of the most spectacular scenery in the UK. It’s a challenging event that will see participants pedal the length of the country over 10 days, averaging almost 100 miles a day.



 

Secondly twelve members of staff from PTC Therapeutics are running the New York Marathon on 1 November 2015 for Action Duchenne. They work for the company across the world in the following countries – Brazil, USA, France, Germany, Switzerland and the UK.

The TCS New York City Marathon is the world’s largest marathon, with more than 50,000 finishers in 2013. It began in 1970 with a small group of runners in Central Park and has been held every year since, with the exception of 2012, when the race was cancelled due to Hurricane Sandy. The course starts in Staten Island and passes through all five boroughs of the city."



'via Blog this'

» German Federal Joint Committee Issues Positive Medical Benefit Rating for Translarna™ in Duchenne Action Duchenne

» German Federal Joint Committee Issues Positive Medical Benefit Rating for Translarna™ in Duchenne Action Duchenne: "PTC Therapeutics, Inc today announced that the Benefit Assessment by Germany’s Federal Joint Committee (G-BA) indicated that Translarna™ (ataluren) provided a benefit for ambulatory patients aged five years and older with nonsense mutation Duchenne Muscular Dystrophy (nmDMD). The G-BA came to the decision that the existing clinical data package presented by PTC provided convincing evidence for the demonstration of a clinically meaningful added benefit. PTC received a 3 in the rating system established under the German pharmaceutical law.

“We are pleased by the positive assessment we received from the G-BA for Translarna. It reflects the medical benefit provided for nonsense mutation DMD patients where there are no other approved therapies,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “We appreciate the G-BA’s recognition of Translarna’s benefit for these patients with such high unmet medical need.”"



'via Blog this'

» Sarepta Therapeutics announce plans to submit rolling NDA for eteplirsen Action Duchenne

» Sarepta Therapeutics announce plans to submit rolling NDA for eteplirsen Action Duchenne: "Sarepta Therapeutics, Inc. announced that the Company held a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding eteplirsen. Sarepta has agreed with the Agency to initiate a rolling NDA submission and will submit the non-clinical: chemistry, manufacturing and controls components of the NDA by the end of this week. As previously announced, Sarepta plans to submit the final component of the NDA by mid-year 2015.
“We will initiate a rolling NDA submission to facilitate the regulatory review of the NDA,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “The initiation of our NDA submission for eteplirsen marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and to continuing to work with the Agency towards the goal of providing treatments to patients as quickly as possible.”
You can read the full press release from Sarepta by opening the document below:"



'via Blog this'

» Action Duchenne submit formal complaint to NHS England. HOW YOU CAN HELP! Action Duchenne

» Action Duchenne submit formal complaint to NHS England. HOW YOU CAN HELP! Action Duchenne: "Action Duchenne have submitted a formal letter of complaint to NHS England over their continued failure to establish a lawful, robust and transparent commissioning service that enables new drugs for the rare disease community to be assessed in an equitable and timely manner.

This letter also draws attention to NHS England’s routine failure to provide families and politicians with clear, accurate and thorough information. This latter point is exemplified by Sir Bruce Keogh (Medical Director of NHS England)’s  recent admission that Individual Patient Funding Requests may be viable route for patients to gain access to Translarna if they will suffer from “imminent, significant and irreversible deterioration” if the drug remains unavailable. This is information families and patients could have benefited from some 10 months ago."



'via Blog this'

BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (NASDAQ:BMRN)

BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (NASDAQ:BMRN): "SAN RAFAEL, Calif., April 27, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen, an investigational exon-skipping drug candidate for the treatment of the largest genetically defined subset of Duchenne muscular dystrophy (DMD). DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with about 20,000 new cases diagnosed globally each year. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients. The company intends to also submit an application for registration in the European Union in summer 2015."



'via Blog this'

Parent Project Muscular Dystrophy Awards $148,000 Grant to University of Washington - PPMD Community

Parent Project Muscular Dystrophy Awards $148,000 Grant to University of Washington - PPMD Community: "Parent Project Muscular Dystrophy (PPMD) has awarded University of Washington a $148,000 grant to continue the functional analysis of spectrin-like repeats in dystrophin. Led by Stanley C. Froehner, PhD, Sackler professor and chair of the Department of Physiology & Biophysics at University of Washington, this grant will provide funding so that more research can be completed before the team submits to the National Institutes of Health (NIH) for additional resources."



'via Blog this'

FOR-DMD Study Webinar (April 2015) - YouTube

FOR-DMD Study Webinar (April 2015) - YouTube: "OR-DMD Study Webinar (April 2015)"



'via Blog this'

» Update on Ataluren (Translarna). Funding deadline in doubt as CPAG meeting is pushed back to June. Action Duchenne

» Update on Ataluren (Translarna). Funding deadline in doubt as CPAG meeting is pushed back to June. Action Duchenne: "Despite consistent assurances from key figures at NHS England, Action Duchenne have learnt that the Clinical Priorities Advisory Group (CPAG) assessment considering the outstanding proposed investments for 2015/16 (including Translarna) is not anticipated to sit until at least June."



'via Blog this'

» Published brief of changes to Disabled Facilities Grant Funding. Action Duchenne

» Published brief of changes to Disabled Facilities Grant Funding. Action Duchenne: "This month heralds a significant change in the way that national funding for disabled facilities grants is paid, and Care and Repair England have published their guidelines. This is aimed for those who, plan, commission and provide health, social care and housing related provision.

In short, instead of national government making direct payment to each local housing authority to help them to meet the cost of providing Disabled Facilities Grants, this allocation will, for the first time, be paid through Better Care Funds. These bodies will then pass on the allocation to the relevant Housing Authority.

To read these guidelines outlining the change in procedure, click the following link:

Disabled Facilities Grant Funding Changes"



'via Blog this'

» Santhera’s Positive Phase III Trial in Patients with DMD Published in The Lancet Action Duchenne

» Santhera’s Positive Phase III Trial in Patients with DMD Published in The Lancet Action Duchenne: "The results of the DELOS trial demonstrated that Raxone/Catena significantly reduced the annual decline in Peak Expiratory Flow (PEF as percent predicted, PEF%p) by 66% compared to patients taking placebo. Other respiratory function endpoints such as Forced Vital Capacity (FVC) and Forced Expiratory Volume (FEV1) corroborated these results and showed a consistent pattern with treatment differences supporting efficacy of Raxone/Catena over placebo in the preservation of respiratory function. Researchers concluded that Raxone/Catena represents a new treatment option for DMD patients."



'via Blog this'

» Santhera Pharmaceuticals receives FDA fast track designation for Raxone/Catena Action Duchenne

» Santhera Pharmaceuticals receives FDA fast track designation for Raxone/Catena Action Duchenne: "Santhera Pharmaceuticals receives FDA fast track designation for Raxone/Catena (idebenone) for the treatment of Duchenne Muscular Dystrophy.

“We are very pleased that the FDA has granted Fast Track designation for Raxone/Catena which further underscores the unmet medical need for effective treatments for patients with DMD” commented Thomas Meier PhD CEO of Santhera. “On the basis of the positive data from our Phase III trial with Raxone/Catena in DMD we have started to prepare a New Drug Application and plan to meet with the FDA in the coming weeks to discuss our NDA dossier in a pre-NDA meeting.”More: http://www.pharmiweb.com/pressreleases/pressrel.asp?ROW_ID=112438#.VSaOvSLF8bU#ixzz3Wp8uen6L"



'via Blog this'

» Researchers Publish Valuable Data on Duchenne Muscular Dystrophy Progression Using the 6-Minute Walk Test Action Duchenne

» Researchers Publish Valuable Data on Duchenne Muscular Dystrophy Progression Using the 6-Minute Walk Test Action Duchenne: "In a recent study titled “Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes,” a research team from the Department of Pediatric Neurology at the Catholic University in Rome, Italy examined the changes at 3 years follow-up in a 6-minute walk test in patients Duchenne muscular dystrophy, the most used primary outcome measure in international multi center clinical trials for patients with the disease."



'via Blog this'

» New exon-skipping strategy rescues dystrophin Action Duchenne

» New exon-skipping strategy rescues dystrophin Action Duchenne: "Antisense oligonucleotides (AONs) hold promise for therapeutic correction of many genetic diseases via exon skipping, and the first AON-based drugs have entered clinical trials for neuromuscular disorders. However, despite advances in AON chemistry and design, systemic use of AONs is limited because of poor tissue uptake, and recent clinical reports confirm that sufficient therapeutic efficacy has not yet been achieved. Here they present a new class of AONs made of tricyclo-DNA (tcDNA), which displays unique pharmacological properties and unprecedented uptake by many tissues after systemic administration."



'via Blog this'

» Breaking News – Translarna Update Action Duchenne

» Breaking News – Translarna Update Action Duchenne: "Contrary to announcements currently circulating, NHS England have provided no guarantees to offer a funding decision on Translarna in May.

Whilst it is true that George Freeman MP and Norman lamb MP have written to NHS England pushing for a decision in May (efforts we wholeheartedly support) Anthony Prudoe, commissioner from NHS England has just announced at the APPG inquiry report launch that there are no guarantees Translarna will be subject to a funding decision in May."



'via Blog this'

PPMD's 2015 Connect Conference - Parent Project Muscular Dystrophy

PPMD's 2015 Connect Conference - Parent Project Muscular Dystrophy: "Parent Project Muscular Dystrophy’s Annual Connect Conference is a unique convergence of industry partners, scientific leaders, medical providers, people living with Duchenne, and their families. This exceptional meeting has grown to be recognized worldwide as the foremost Duchenne muscular dystrophy meeting. But more than that, it is a way for families affected by Duchenne to connect with each other. To build support networks. To realize no one is on their own in the fight to end Duchenne."



'via Blog this'

Spring Forward: Bringing the MD-CARE Act to Life - PPMD Community

Spring Forward: Bringing the MD-CARE Act to Life - PPMD Community: "Despite great odds in a political climate that was stacked against us – we achieved what was considered to have been impossible. After months of outreach to congressional champions and visits to Capitol Hill, the MD-CARE Act Amendments were passed and signed into law in the end of 2014.
Now what? Well… now the real work of implementation actually begins.
But the truth is that our community has so successfully built champions and partners across the federal agencies over the last two decades, that the implementation of the MD-CARE Amendments started long before the bill ever passed. Our federal partners ‘anticipated’ its passage and began to implement aspects of what was being called for in the new law, even before Congress was telling them they had to."



'via Blog this'

What is Epicatechin? - PPMD Community

What is Epicatechin? - PPMD Community: "Epicatechin is a flavonoid found in dark chocolate harvested from the cacao tree. What’s that got to do with Duchenne? Epicatechin is one of dozens of flavonoids found in dark chocolate, but it is unique with respect to its effects on muscle and heart. Epicatechin has been demonstrated in animals and humans to increase the production of new mitochondria in heart and muscle (termed “mitochondrial biogenesis”) while concurrently stimulating the regeneration of muscle tissue. The progression of muscle weakness in Duchenne muscular dystrophy is associated with a loss of mitochondria, which power the skeletal muscle. Recent evidence indicates that Epicatechin is acting as a mimic of a newly discovered human hormone released by normal muscles after exercise that may be responsible for mitochondria biogenesis and stronger muscle fibers that occur normally after exercise training. Other closely related flavonoids in chocolate either have no effect or are actually inhibitory to mitochondrial biogenesis. Thus use of extracts of cacao or non-pharmaceutical grade Epicatechin may be ineffective. The current studies in muscular dystrophy are being conducted with pharmaceutical grade Epicatechin, free of any other flavonoids or contaminants. There is a trial in Europe evaluating a related compound, epigallocatechin gallate (EGCG) for its anti-oxidant effects in muscular dystrophy. ECGC has none of the mitochondrial or muscle effects that has been observed for Epicatechin, because it is the wrong shape to mimic the human hormone."



'via Blog this'

Recall Notice for Deflazacort 30mg - PPMD Community

Recall Notice for Deflazacort 30mg - PPMD Community: "PPMD has been in touch with Masters Pharmaceuticals regarding a recall that was sent to many families regarding a recent shipment of Deflazacort. Here is what they told us:
 
The recall is specific to Deflazacort 30mg, (Marketing Authorisation Holder: Sandoz) and was recently issued by the Spanish Agency of Medicine. The batches that are affected and subject to the recall notification are J021 (expiry 30/06/2017) and J028 (expiry 30/09/2017). Masters is currently trying to establish the reason for the recall but have no further information at this time, but they are working with the Products manufacturer. They are contacting ~230 customers who purchased the affected batches however, we believe that most will only have batch J028 remaining."



'via Blog this'

Important New Duchenne Steroid Protocol: Download the "PJ Nicholoff Steroid Protocol" - PPMD Community

Important New Duchenne Steroid Protocol: Download the "PJ Nicholoff Steroid Protocol" - PPMD Community: "Phillip James “PJ” Nicholoff was a vibrant, 31-year-old man living with Duchenne muscular dystrophy. He had an encyclopedic love of all college and professional sports; he and his family traveled all over the country to catch games. He had a degree in web design and worked his entire adult life. He enjoyed swimming, dating, and driving his own car – much like any other 31-year-old young man.
 
PJ had been treated with daily corticosteroids since the age of 6. He endured several pathologic fractures, likely a result of his steroid treatment and his non-ambulatory status. In November 2013, on his last trip to Florida, he fractured his humerus and hip, and was transported by plane to a hospital closer to his northern home. PJ had orthopedic surgery to manage both of these fractures. After these repairs, he developed respiratory distress, shock, and died. Review of the medical record suggested that he had not received consistent corticosteroid replacement during his hospitalizatio"



'via Blog this'

Parent Project Muscular Dystrophy & Foundation to Eradicate Duchenne Award $1 Million Grant to ReveraGen BioPharma - PPMD Community

Parent Project Muscular Dystrophy & Foundation to Eradicate Duchenne Award $1 Million Grant to ReveraGen BioPharma - PPMD Community: "Parent Project Muscular Dystrophy (PPMD) is excited to join forces with Foundation to Eradicate Duchenne (FED) to award ReveraGen BioPharma (ReveraGen) a $1 million grant to help fund the chronic toxicology program for VBP15, a combined action drug that addresses multiple levels of Duchenne."



'via Blog this'

Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask) - PPMD Community

Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask) - PPMD Community: "This webinar will review the basic biology of dystrophin (the gene, structure-function of the protein, its role in the dystrophin-glycoprotein complex, characteristics of different forms of dystrophin (both naturally occurring and drug-induced truncated forms), discuss how dystrophin is measured in both preclinical studies and clinical trials, and discuss the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. We will also discuss what is required in order to have dystrophin considered as a surrogate."



'via Blog this'

Duchenne Muscular Dystrophy - Summit

Duchenne Muscular Dystrophy - Summit: "Our utrophin modulation programme is developing oral, small molecule drugs that increase the production of a protein call utrophin.  Utrophin is a naturally occurring protein that is functionally and structurally similar to dystrophin.  Utrophin is produced during the early stages of muscle fibre development but is switched-off in maturing muscle fibres, at which point dystrophin is produced to perform the same functional role.  When a muscle fibre is damaged, utrophin is also produced during the early stages of the repair mechanism.

Our utrophin modulation approach aims to use small molecule drugs to maintain the production of utrophin to compensate for the absence of dystrophin in DMD patients and so protect healthy muscle function.  A significant advantage of utrophin modulation is that it is independent of the underlying genetic fault and therefore has the potential to treat 100% of DMD patients.  We also believe it could be complementary to other DMD treatment approaches.

The concept of utilising utrophin as a treatment for DMD was developed by our co-founder and scientific advisor Professor Kay Davies at the University of Oxford."



'via Blog this'

Researchers generate mature, functional skeletal muscles using new approach

Researchers generate mature, functional skeletal muscles using new approach: "A team of researchers from Italy, Israel and the United Kingdom has succeeded in generating mature, functional skeletal muscles in mice using a new approach for tissue engineering. The scientists grew a leg muscle starting from engineered cells cultured in a dish to produce a graft. The subsequent graft was implanted close to a normal, contracting skeletal muscle where the new muscle was nurtured and grown. In time, the method could allow for patient-specific treatments for a large number of muscle disorders. The results are published in EMBO Molecular Medicine."



'via Blog this'

» Eplerenone improves cardiac function in Duchenne patients Action Duchenne

» Eplerenone improves cardiac function in Duchenne patients Action Duchenne: "A recent study showed that a specific cardiac drug is able to induce cardiac function improvement in boys with muscular dystrophy and cardiomyopathy. The study is entitled “Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomized, double-blind, placebo-controlled trial.”

“With increasing recognition of the effect of cardiomyopathy on morbidity and mortality in Duchenne muscular dystrophy, improved strategies are needed to change the natural history of declining left ventricular systolic function and to attenuate its sequelae,” wrote the research team.

Eplerenone is a cardiac medication, used alone or in combination with other drugs to treat high blood pressure. In this study, the authors evaluated the efficacy of eplerenone in patients suffering from DMD and early myocardial disease.Interestingly, in patients taking eplerenone, a significant decrease in the decline in cardiac function was only observed after at least 6 months of therapy.

“Our findings suggest that early treatment with eplerenone attenuates decline in cardiac function, which is a leading cause of death in Duchenne muscular dystrophy,” wrote the research team."



'via Blog this'

Parent Project Muscular Dystrophy Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne - PPMD Community

Parent Project Muscular Dystrophy Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne - PPMD Community: "PPMD announced today that they will award Dr. M. Carrie Miceli and her team at UCLA’s David Geffen School of Medicine and College of Letters and Science, a $50,000 exploratory grant to continue their evaluation of exon skipping boosters for the treatment of Duchenne. 
 
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500 to 5,000 live male births. The loss of a key muscle protein called dystrophin causes muscle wasting and weakness, eventually leading to the loss of ambulation, difficulty breathing, and heart failure.  Death typically occurs in the mid-to late 20s.
 
Duchenne is often caused by frame-shifting mutations that abolish dystrophin expression that can be repaired by antisense oligonucleotide (AON) directed “exon skipping.” Ongoing clinical trials of exon 51 AON demonstrate dystrophin rescue and slowing of disease progression. Adding a targeted small molecule drug, in combination with the AON, may be a means of increasing skipped dystrophin levels and thereby increasing the effectiveness of treatment with AON alone. Dr. Miceli and her team at UCLA have identified drugs that boost AON-directed exon 51 skipping, which impinge on a common pathway. This has led her to predict that second-generation drugs may have even greater skip boosting activity. This grant will allow Dr. Miceli to continue exploring therapies that will promote skipping alone or in combination with AON directed against exon 51 or other exons currently in the clinical pipeline."



'via Blog this'

» Tricyclo-DNA for Duchenne Action Duchenne

» Tricyclo-DNA for Duchenne Action Duchenne: "Recently, researchers from Bern developed an active substance, which together with an international team they tested successfully. This promising therapy approach is to correct the defect in the production of dystrophin with short segments of a chemically modified DNA, so-called antisense oligonucleotides (AO). A significant curative effect with the tested active substances had not been achieved until now. That is because the corresponding active substances are not yet active enough and do not reach vital muscles such as the heart.

 

Experiments in mice show evidence that the agent leads to an improved dystrophin production in all muscles including the heart and the lungs and subsequently leads to an improved mobility and an increased life expectancy in mice.

 

Surprisingly it was also observed that the dystrophin production in the brain was corrected as well. It was thus shown for the first time that an oligonucleotide can pass the blood-brain barrier and become active there.

 

The next step of the clinical trial is to test tricyclo-DNA in humans. The trial is coordinated by the spin-off enterprise Synthena AG of the University of Bern founded in 2012. The enterprise manufactures tricyclo-DNA and advances the development of a medication for Duchenne patients.

 "



'via Blog this'

» Cardiac drugs show cardioprotective benefit in boys with Duchenne Action Duchenne

» Cardiac drugs show cardioprotective benefit in boys with Duchenne Action Duchenne: "“Our findings suggest that early treatment with eplerenone attenuates decline in cardiac function, which is a leading cause of death in Duchenne muscular dystrophy,” the researchers wrote. “Although the fundamental goal of this study was to test the use of early intervention with available drugs in a disorder in which patients are likely to have progressive cardiomyopathy, the need for long-term clinical follow-up is apparent when recognizing that hard events, such as cardiac failure and death, are unlikely to occur for many more years.”"



'via Blog this'

Important Vaccinations Recommendations - PPMD Community

Important Vaccinations Recommendations - PPMD Community: "Vaccinations are very, very important to protect us against infection and disease – but they are also very, very confusing. 
Some vaccines are “live,” some are “attenuated (killed),” some protect against viral infections, some protect against bacterial infections, some are given only in childhood, some need a “booster” later in life, some are only given later in life…. Adding Duchenne – medications, pulmonary, and cardiac function – to the picture adds even more confusion.
 
In order to answer the regularly asked questions, “to give or not to give, when to give, how to give and what to give,” we have teamed up with the Center for Duchenne Muscular Dystrophy (CDMD) at UCLA to provide the following recommendations. A huge “thank you!” to Drs. Stan Nelson, Paul Krogstad, and Carrie Miceli, (UCLA) for their contributions in developing this document. 
 "



'via Blog this'

» Marathon Pharmaceuticals announce FDA fast track designation for deflazacourt Action Duchenne

» Marathon Pharmaceuticals announce FDA fast track designation for deflazacourt Action Duchenne: "Marathon Pharmaceuticals, LLC, a biopharmaceutical company focused on developing treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for deflazacort in the treatment of patients with Duchenne Muscular Dystrophy (DMD). Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties."



'via Blog this'

Heart Failure Drugs Slow Deadly Damage in Duchenne, Offering New Hope - PPMD Community

Heart Failure Drugs Slow Deadly Damage in Duchenne, Offering New Hope - PPMD Community: "Less heart muscle damage was seen at 6 months, and less decline in heart function was seen at 12 months when adding eplerenone to background ACE inhibitor or ARB therapy compared to adding placebo. The research team believes that by benefiting heart function at a very early stage, longer-term complications of cardiomyopathy such as heart failure can be greatly reduced. While continued research is needed to develop even better treatments for tomorrow, the availability of these medicines offers boys with Duchenne something beneficial for their hearts today."



'via Blog this'

Update on the 2015 Flu - PPMD Community

Update on the 2015 Flu - PPMD Community: "What should I do if I think my child has the flu?
 
If your child has symptoms of the flu, call your primary health care provider immediately!  Rapid diagnostic testing, which checks secretions in the throat for the influenza virus, is 50-70% accurate for diagnosing influenza (most providers will send a negative specimen for culture, just to be sure that there is no virus present).  If the test is positive, starting an antiviral medication is recommended.  Antiviral medication should be started as soon as possible in the course of the illness and continued for 5 days.   It is best if this medication is started within 48 hours of the beginning of symptoms, but the treatment can still has some benefit if started later, especially for patients who are experiencing a longer or more complicated course."



'via Blog this'

Tivorsan Pharma Meets Development Milestones with DMD Treatment Research

Tivorsan Pharma Meets Development Milestones with DMD Treatment Research: "Tivorsan Pharmaceuticals, Inc. (Tivorsan), a protein therapeutics company with a focus on developing treatments for neuromuscular disorders such as muscular dystrophy, recently announced the company has achieved a fourth set of pivotal milestones under a series of research grants from Parent Project Muscular Dystrophy (PPMD) and Muscular Dystrophy Association (MDA). These achievements mark the release of more funding from these grants, in fulfillment of PPMD’s pledge of $565,000, and the MDA’s pledged $1 million."



'via Blog this'

Tivorsan Pharmaceuticals Achieves Key Development Milestones under its Parent Project Muscular Dystrophy and Muscular Dystrophy Association Awards | Business Wire

Tivorsan Pharmaceuticals Achieves Key Development Milestones under its Parent Project Muscular Dystrophy and Muscular Dystrophy Association Awards | Business Wire: "PROVIDENCE, R.I.--(BUSINESS WIRE)--Tivorsan Pharmaceuticals, Inc. (Tivorsan) today announced that it met its fourth (4th) set of critical milestones under its existing grants from Parent Project Muscular Dystrophy (PPMD) and Muscular Dystrophy Association (MDA). The achievement of these milestones triggers additional payments to Tivorsan pursuant to the Company’s $565,000 grant from PPMD and $1,000,000 grant from MDA."



'via Blog this'

Early use of heart failure drugs slows cardiac damage in boys, young men with DMD

Early use of heart failure drugs slows cardiac damage in boys, young men with DMD: "Early use of available heart failure drugs slows the progressive decline in heart function before symptoms are apparent in boys and young men with Duchenne muscular dystrophy (DMD), according to a new study published online by The Lancet Neurology."



'via Blog this'