Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.
Apilimod mesylate by OrphAI Therapeutics for Amyotrophic Lateral Sclerosis: Likelihood of Approval
Apilimod mesylate is under clinical development by OrphAI Therapeutics and currently in Phase II for Amyotrophic Lateral Sclerosis.