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 […]
I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe. Like you, I am heartbroken and […]
The wait continues. Sarepta Therapeutics announced this morning that the FDA will not complete the review of the eteplirsen new drug application by the PDUFA date. Considering the thousands […]
NORD hosted the 2016 Rare Impact Awards to bring together the rare disease community and to honor and celebrate outstanding achievements. Last night, Debra Miller, founder of CureDuchenne, was […]
From one mom to another, I wish you a Happy Mother’s Day from the bottom of my heart. As I think about the most wonderful gift in my life, […]
CureDuchenne, the California-based nonprofit organization dedicated to finding a cure for every person with Duchenne muscular dystrophy, has released the following statement from its co-founder and CEO, Debra Miller, […]
As I prepare to travel to Monday’s Advisory Committee meeting I continue to be disappointed by the FDA Peripheral and Central Nervous System Drugs Advisory Committee’s initial review of […]
For those who battle against Duchenne muscular dystrophy, there is more hope now than ever. Recent medical breakthroughs in the form of effective drugs are slowing the disease’s progression […]
