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Pat Furlong of Parent Project Muscular Dystrophy kindly shared this whitepaper, ‘Benefit-Risk Assessments in Rare Disorders: The case for therapeutic development
Total Orphan Drugs covers PPMD's whitepaper.
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Preliminary Clinical Data from Ongoing Phase 1b/2a Clinical Program Presented at New Directions in Biology and Disease of Skeletal Muscle 6th Biennial Conference Cambridge, Mass.—July 3, 2014—Akashi Therapeutics, Inc., a...
Prosensa Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval More good news from the FDA! Th…
EMA grants Conditional Approval for Ataluren Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional Approval. This is a big moment for Du…
Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional Approval. This is a big moment for Duchenne, a landmark decision and a sign of hope for all. While Ataluren is targeted at a small subset of patients (13% +/-), the conditional approval is a sign that the EMA worked very hard to understand Duchenne, that they recognize this unmet need, the progressive and debilitating process of Duchenne and the urgent need to treat.
Great Great day! Agree!
Below you can find the latest update from Prosensa regarding a potential regulatory path forward for drisapersen.Regulatory discussions ongoing & re-do…
Sarepta Therapeutics Announces Agreement for Acquisition of Manufacturing Facility in Massachusetts
State-of-the-art 60,000 square foot facility enhances internal manufacturing capability
Re-dosing plans are underway and 96-week data from an open-label extension study of drisapersen for the treatment of DMD presented at the American Academy of Neurology (AAN)
As researchers embark on a project to connect 30 million patient records, questions about privacy arise.
Commentary by Eric Hoffman and Beth McNally.
In the first international study of its kind, researchers have found that there are many different costs accompanying a rare condition such as Duchenne muscula…
From BioPortfolio: Akashi Therapeutics, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the companyâ��s most adv...
After a late-stage failure and a kiss-off from partner GlaxoSmithKline, Prosensa and its innovative treatment for Duchenne muscular dystrophy seemed to be on a downward slope.
Two DMDRP PAs have been posted on the CDMRP website.
Read the press release from PTC Therapeutics: The first treatment for the underlying cause of Duchenne muscular dystrophy SOUTH PLAINFIELD, NJ – May 23, 2014…
This webinar will serve to answer several key questions: - How do we balance the need for timely access new medicines and benefit risk? - Who ultimately should chose what level of uncertainty is acceptable? - What are good examples of tools and methods for making informed benefit risk decisions with increased uncertainty? Featuring: - Andrea Beyer, National Expert Benefit Risk Assessment, European Medicines Agency - Yann Le Cam, Chief Executive Officer at EURORDIS - Alastair Kent, Director of Genetic Alliance UK - Donald Singer, Secretary, European Association of Clinical Pharmacology and Therapeutics
In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profil…
In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profile in twelve boys with Duchenne. Plasma levels of the drug were variable and the company speculates that diet and the course of the disease may be the cause.
The preliminary trial data will be reviewed further by Summit and is expected to lead to a revision of future clinical trial plans in order to determine the optimal way, either through dietary means or drug formulation changes, to address the drug uptake differences between Duchenne patients and healthy volunteers. The next patient study is now expected to start in Q4 2014.
Summit will host a webinar next week to discuss this news and answer your questions - more details to come. For now though, you can submit your questions to firstname.lastname@example.org, RE: SUMMIT.
The latest update from Prosensa regarding re-dosing drisapersen and the regulatory path forward.
Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, today announced that Michael S. Wyzga has been nominated for appointment to its Supervisory Board at the next shareholders' meeting, scheduled for June 17, 2014.