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.
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.
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...
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.