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NORD Rare Impact Awards
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 […]
WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in Duchenne Muscular Dystrophy
Happy Mother’s Day
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 Responds to FDA Advisory Committee’s Recommendation Against Approval of Eteplirsen for Duchenne Muscular Dystrophy
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, […]
Sarepta Advisory Committee Meeting
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 […]
Supporting Sarepta, Providing Hope
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 […]
NICE Recommends Translarna™ (ataluren) for the Treatment of Patients with Nonsense Mutation Duchenne Muscular Dystrophy in England