Remove tag ataxia
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HP&M Attorneys Receive Accolades: Top Lawyer Under 40 and WWL: Life Sciences 2023 Global Elite Thought Leader

FDA Law Blog

Duchenne, Friederichs’s Ataxia, Sickle Cell Disease, Chagas, ALS). In addition to Mr. Karst, ten other HP&M Directors were tagged as “Recommended” by WWL: Life Sciences 2023: Ricardo Carvajal Robert A. Dormer Jeffrey N. Gibbs Paul M. Hyman Alan M. Kirschenbaum Allyson B. Mullen Frank J. Sasinowski Jeffrey K.

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New Rare Disease Drugs and Research Advancements

XTalks

Skyclarys (omobaloxone) was approved in 2023 for the treatment of adults and pediatric patients 16 years of age and older with Friedreich’s ataxia, a rare, neurological disease​​. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.