Credit: Getty Images. Laura Buch, MSPAS, PA-C, emphasizes the need for newborn screening to detect infantile-onset lysosomal storage diseases and swift treatment. In honor of PA Week 2024, we ...
For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. Friday, the FDA approved Zevra’s arimoclomol capsules to treat the rare lysosomal storage ...