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.

News Release | sareptatherapeutics.com

News Release | sareptatherapeutics.com: CAMBRIDGE, MA -- (Marketwired) -- 10/02/13 -- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced a new online resource center, called Let's Skip Ahead, for families affected by Duchenne muscular dystrophy (DMD) and their healthcare providers. The new website, available at www.skipahead.com, includes information and educational resources intended to make the science of exon skipping simple, and provides an opportunity to sign up for updates about upcoming Sarepta clinical trials.

http://www.skipahead.com/

News Release | sareptatherapeutics.com

News Release | sareptatherapeutics.com: Sarepta Therapeutics Announces Eteplirsen Demonstrates Continued Stability on Walking Test Through 96 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced data through Week 96 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results through nearly two years showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT). As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at Week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes.

After 96 weeks, patients in the 30 mg/kg and 50 mg/kg eteplirsen cohorts who were able to perform the 6MWT (modified Intent-to-Treat or mITT population; n=6) experienced less than a 5 percent decline (17.5 meters) from baseline in walking ability. A statistically significant treatment benefit of 70.8 meters (p ≤0.001) was observed for the mITT population compared with the placebo/delayed-treatment cohort (n=4), which initiated treatment at Week 25 following 24 weeks of placebo. After experiencing a substantial decline earlier in the study, the placebo/delayed-treatment cohort also demonstrated stabilization in walking ability from Week 36 through 96, the period from which meaningful levels of dystrophin were likely produced, with a decline of 18.5 meters over this timeframe. These analyses were based on the maximum 6MWT score when the test was performed on two consecutive days.

"We are very encouraged the study has demonstrated walking stability in patients for more than a year since confirming that eteplirsen treatment produced dystrophin in their muscles," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We look forward to sharing these updated data with the FDA as part of our New Drug Application for eteplirsen, which we plan to submit in the first half of 2014."

All Party Parliamentary Group - Access to high-cost drugs for rare diseases - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

All Party Parliamentary Group - Access to high-cost drugs for rare diseases - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Yesterday, Paul Lenihan – our CEO attended the Parliamentary reception briefing on the launch of the APPG’s report into access to high-cost drugs for rare diseases. Hosted and introduced by Dave Anderson MP, Action Duchenne endorses the four main recommendations contained within the report. These call for: 

1. The Government to establish ring-fenced funds for rare disease drugs. 

2. National Institute for Clinical Excellence (NICE) to access treatments for rare conditions in a different way from less rare conditions 

3. NICE and NHS England to speed up access to life-changing drugs after final stages of clinical trials to ensure there are no major delays in treatments reaching children. 

4. NHS England to ensure specialist centres are equipped with an appropriate range of health professionals to deliver treatments.