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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 […]
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 […]
Giving our Sons with Duchenne the Best Care Possible
When our son, Hawken, was diagnosed 13 years ago, we were told to go home and love your son…there is nothing you can do. Those doctors were wrong, […]
Why PTC Withdraws Regulatory Filing in Canada
PTC withdraws the regulatory filing in Canada so they can supplement the phase II data with the phase III, ACT-DMD data. Why is PTC withdrawing the NDS from Canada? […]
PTC Therapeutics Patient/Parent Call
Stu Peltz from PTC Therapeutics hosted a call today regarding the Refuse to File letter from the FDA, dated February 22nd. More detailed information is available in the transcript […]
Rare Disease Day
Today, the last day in February, is Rare Disease Day when the world pauses for a moment to focus on often overlooked diseases that affect relatively few people but […]