In this webinar, the NS Pharma team presents updates for the Duchenne community: Access programs for Viltepso, which is suitable for individuals with Duchenne who are amenable to exon 53 […]
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 in Participants With Duchenne Muscular Dystrophy Sarepta is now expanding their trial of SRP-9001 (ENDEAVOR) to […]
This presentation by Lauren Morgenroth, CGC and Ana Christensen, MPH at TRiNDS is part of the “A Holistic View of Clinical Trials” webinar and covers the following topics: the clinical […]
Dear Friends, We are pleased to provide you with detailed information from Santhera Pharmaceuticals about an ongoing clinical trial that your son may be able to participate in. Beyond investing […]
Dr. Gilmore O’Neill, Chief Medical Officer of Sarepta Therapeutics, Inc, gave a scientific overview of Sarepta’s RNA-targeted exon skipping technologies, PMO and PPMO. Dr. O’Neill provided valuable information on this […]
CureDuchenne hosted a live Twitter chat on September 20 to raise awareness and address the important issues facing the Duchenne community. It was an opportunity to hear from patients, parents, […]
Dr. Kevin Flanigan from Nationwide Children’s Hospital provided an update on his Dup2 research during a CureDuchenne hosted call with families on September 6. Back in 2010, Dr. Flanigan told […]
Approach: Cardiac stem cell derived therapy
Funding Impact: Initiation of Phase 1/2 study (HOPE Clinical Trial)
Successful Exit: Stock Return
As a parent of a son with Duchenne I have been frustrated that the data presented to the FDA does not match the patient experience. I personally know so […]
