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 […]
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 […]
Guest blog by Tammy and Scott Henegar Our family’s journey with Duchenne muscular dystrophy started in March 2011 when our 4 ½ year old adventurous and sweet young boy […]
My husband Paul and I have dreamed of this day since our son Hawken was diagnosed with Duchenne almost 15 years ago! We are overwhelmed with joy to announce […]
BioMarin is providing a further update to the Duchenne Community following our announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of […]
Gene therapy proposes a promising approach in treating Duchenne, placing corrected genes into cells that have missing or damaged genes. Although gene therapy is still relatively early, CureDuchenne is […]
We are sharing the following update from BioMarin. We would like to update the patient community in light of the recent announcement regarding the EMA marketing application. BioMarin is […]
