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Rare disease spotlight: treating Friedreich's ataxia

Rare disease spotlight: treating Friedreich's ataxia
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Nguengang Wakap , Sylvia Boesch , Toby Ferguson , University Of Pennsylvania , European Commission , Rare Movement Disorders Innsbruck , Head Of The Center , Head Of The Neuromuscular Development Unit , Committee For Medicinal Products Human Use , Department Of Neurology , University Of Florida , University Innsbruck , Reata Pharmaceuticals , Vice President , Neuromuscular Development Unit , Medicinal Products , Human Use , Rare Disease Day , Principal Investigator , Medical University Innsbruck , Friedreich Ataxia Rating Scale , Phd Toby , Eurj Med ,

First treatment for Friedreich's ataxia in Europe granted approval

Biogen Inc. has taken a step closer to bringing its ‘groundbreaking treatment’ to patients with a rare, genetic, life-shortening, debilitating, and neurodegenerative disorder. ....

Sylvia Boesch , Priya Singhal , Rare Movement Disorders Innsbruck , Department Of Neurology , European Union , Friedreich Ataxia Research Alliance , European Commission , European Medicines Agency , University Innsbruck , Medical University Innsbruck , Friedreich Ataxia Rating Scale , Chief Executive Officer , Ataxia Research Alliance , Markets Amp Regulations , Clinical Development , Atient Centricity , Pis Active Pharmaceutical Ingredients , Hasei Ii , Rare Disease , European Commission ,