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PTC THERAPEUTICS RECEIVES POSITIVE OPINION FROM CHMP FOR TRANSLARNA™ (ATALUREN)
PTC Therapeutics announced this morning that they have received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency, regarding the company’s […]
PTC THERAPEUTICS RECEIVES POSITIVE OPINION FROM CHMP FOR TRANSLARNAâ„¢ (ATALUREN)
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company’s application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.
Prosensa Update: Regulatory Discussions Ongoing & Re-dosing on Track
Dear Patient Group Representative, We trust this letter finds you well. As announced in a press release and during our quarterly update for the financial community […]
Summit Report Preliminary Results from Phase 1b Clinical Trial of SMT C1100 for DMD
SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease.
The Phase 1b trial was a dose-escalating, open-label study conducted in pediatric patients with DMD to evaluate safety, tolerability and drug exposure.
CureDuchenne Founder Debra Miller is a Panelist at the California Women’s Conference
CureDuchenne Founder and CEO Debra Miller is a panelist at the California WomenÌ¢âÂã¢s Conference on May 19 at the Long Beach Convention Center. Miller will participate in a panel called Ì¢âÂÒA New Approach to Non-Profit Impact.Ì¢âÂÌ_ She will speak about venture philanthropy and how CureDuchenne has created a successful business model to help accelerate Duchenne research.
Blingo to CureDuchenne Raised $175,000 for Muscular Dystrophy Research
CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, held its inaugural Blingo to CureDuchenne event on May 3 at the Hyatt Regency in Philadelphia. Nearly 300 people, including PhiladelphiaÌ¢âÂã¢s top business and community leaders, enjoyed an elegant evening of bingo and bling with celebrity guests while raising more than $175,000 to support the most promising research projects aimed at treating and curing Duchenne.
Santhera Announces Successful Outcome of Phase III Study with Catena®/Raxone® in Duchenne Muscular Dystrophy
Santhera Pharmaceuticals (SIX: SANN) announces today that its Phase III DELOS study of orally administered Catena®/Raxone® (INN: idebenone) in patients with Duchenne Muscular Dystrophy (DMD) met the primary endpoint and achieved its primary objective of delaying the loss of respiratory function compared to placebo.
EY Announces CureDuchenne Founder and CEO Debra Miller is an Entrepreneur of the Year 2014 Award Finalist in Orange County
EY today announced that Debra Miller, founder and CEO of CureDuchenne, is a finalist for the EY Entrepreneur Of The YearÌ¢Û_å¢ 2014 Award in Orange County. The awards program recognizes entrepreneurs who demonstrate excellence and extraordinary success in such areas as innovation, financial performance and personal commitment to their businesses and communities. Miller was selected as a finalist from nearly 80 applications by a panel of independent judges. Award winners will be announced at a special gala event on June 4, 2014 at The St. Regis Resort in Dana Point.