Anaphylaxis and Desensitization to Alglucosidase Alfa in late-onset Pompe Disease ten years later after initial enzyme replacement therapy administration: a possible drug-drug interaction of CGRP inhibiting therapies

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Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy (EMBARK): Pivotal Phase 3 Primary results

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A Pilot Phase 2 Randomized Trial to Evaluate the Safety and Potential Efficacy of Etravirine in Friedreich Ataxia Patients

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RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)

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Rescue from COVID-19 infection after precipitation of MG crisis in late-onset myasthenia gravis

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