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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 Provides Update on Health Canada Review of Translarna™ (ataluren) for the Treatment of Duchenne Muscular Dystrophy
Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)
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 […]