ANAHEIM, Calif., Nov. 15, 2017 /PRNewswire/ — Boys and young men in advanced stages of Duchenne muscular dystrophy experienced significant and sustained improvements in cardiac structure and function, as well […]
Funds to support continued development of SingleCut CRISPR technology for Duchenne muscular dystrophy CAMBRIDGE, Mass. – November 8, 2017 – Exonics Therapeutics, Inc., a biotechnology company focused on developing SingleCut CRISPR […]
Dr. Eric Olson to Provide CRISPR/Cas9 and Gene Therapy Update During November 16 Webinar We are delighted that Exonics Therapeutics has closed a $40 million Series A financing with […]
No matter how long we live, the ones who will have known us the longest are our siblings. For siblings Wil and Emily Cook, the time they have together is […]
Philadelphia, PA (October 31, 2017) – Philadelphia celebrities and more than 250 prominent leaders, community members and guests raised $240,000 to fund Duchenne muscular dystrophy research at the fourth annual […]
PTC Therapeutics held a community conference today regarding Ataluren. Stuart Peltz, President and CEO, PTC Therapeutics, Inc., welcomed and thanked the community as well as […]
NEWPORT BEACH, Calif., October 26, 2017 – CureDuchenne, a nonprofit dedicated to funding research and improving patient care for Duchenne muscular dystrophy, launched the CureDuchenne Cares Virtual Durable Medical Equipment […]
Dear Duchenne Community, As you might have seen, the Office of Drug Evaluation I of the FDA provided a Complete Response Letter (CRL) for the application to market ataluren in […]
PTC Therapeutics received a Complete Response Letter from the Food and Drug Administration (FDA) for Ataluren’s New Drug Application (NDA). This means that the FDA has completed its review of […]
