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."



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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."



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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.
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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. "



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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.