RallyBio Avenzo merger aims to strengthen rare disease pipeline after a previous attempt with Candid Therapeutics fell through.
Author: PharmaSignal News Desk
Servier Edgewise acquisition involves a muscular dystrophy tablet with potential peak sales exceeding $2 billion.
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Regenxbio Duchenne gene therapy aims for FDA approval despite side effects.
• Regenxbio seeks FDA approval for Duchenne gene therapy.
• Therapy targets rare disease affecting 1 in 3,500 boys.
• CEO optimistic despite two serious side effects.