Here are top line notes from the PRO044 presentation at World Muscle Society, Oct 4. Age of inclusion: 5-16 Mean age: 9 The drug was well tolerated. They saw trace […]
Conference call and live webcast scheduled for 11:00 a.m. ET (5:00 p.m. CET) Tuesday, October 8, 2013 LEIDEN, The Netherlands – October 3, 2013 – Prosensa Holding N.V. (NASDAQ: RNA), […]
We wanted to let Duchenne families know about a new online resource on exon skipping from Sarepta Therapeutics. Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today […]
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.
We are encouraged by the promising Phase IIb data for eteplirsen, an exon-skipping compound, which we hope could be a breakthrough treatment for Duchenne muscular dystrophy. Currently, there is no […]
CureDuchenne announced today the launch of CureDuchenne Ventures, a new initiative to identify and develop a robust pipeline of therapies to treat Duchenne, the most common and lethal form of muscular dystrophy. This drug discovery and development initiative is based on CureDuchenneÌ¢âÂã¢s successful venture philanthropy model, in which the organization provides early stage funding to biotechnology, pharmaceutical companies and academic investigators to help develop breakthrough therapies to treat Duchenne.
CureDuchenne raised more than $300,000 for Duchenne research at the third annual Getzlaf Golf Shootout hosted by Ryan Getzlaf, captain of the Anaheim Ducks. The event, held on September 7 and 8, benefited CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy. Getzlaf was joined by current and former Anaheim Ducks players including Teemu Selanne, Corey Perry, Dustin Penner, Andrew Cogliano, Cam Fowler and others who teamed up with each foursome.
GlaxoSmithKline (GSK) and Prosensa today announced that GSK’s Phase III clinical study of drisapersen, an investigational antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy (DMD) patients with an amenable […]
Background: GlaxoSmithKline (GSK) and Prosensa Therapeutics today announced the results of Phase III clinical data for drisapersen, an exon-51 skipping compound for the treatment of Duchenne muscular dystrophy. The data […]
