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Türkiye · TurkeyThe steroid therapy Agamree (vamorolone), which is used for helping to preserve muscle function in people with Duchenne muscular dystrophy (DMD), will be sold and promoted in Turkey by Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. (GEN), per an agreement with Santhera Pharmaceuticals. “As leaders in the Turkish specialty pharmaceuticals market, GEN were a natural choice for Santhera as we looked for a high-quality distribution partner,” Dario Ek…Read Article
Duchenne MD therapy Agamree to be sold in Turkey starting in 2026
Mississippi · MississippiI’m a musician, and so are my brothers, Ron and Tim. We’ve all played professionally, but it’s been a long time since we’ve played together. That changed last month, but let me explain why it was such an event. Back in the late ’70s we were part of the Frank Kalik Orchestra, a Polka Hall of Fame group based in Pittsburgh that did some great music. Looking for something different, I left the polka band to join a Top 40 group. A couple of years la…Read Article
Celebrating a long-in-the-works family music songfest
FDA · United StatesThe U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Dyne Therapeutics’ DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. This designation is intended to accelerate the development and regulatory review of medications intended to treat serious conditions. Benefits include enhanced FDA support in development and decision-making, regular communication wi…See the Story
