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Neurology and Therapy|April 27, 2025
Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in Australian HospitalsIan R Woodcock, Didu S Kariyawasam, Maina P Kava, et al.
Neurology and Therapy|November 10, 2025
Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N-of-1 Trial with 21 months Clinical ObservationAndrew Cook, Beth Stuart, Antonio Ochoa-Ferraro, et al.
Neurology and Therapy|October 28, 2025
One Size Does Not Fit All: Novel Individualized Use of Dexamethasone in Infantile Epileptic Spasms SyndromeEkaterina Kabak, Isabella Claudia Leitner, Birgit Stark, et al.
Neurology and Therapy|October 14, 2025
Summary of Research: Fertility Outcomes in Risdiplam-Treated Male Patients with Spinal Muscular Atrophy: A Multicenter Case SeriesShelley Coskery, Marcus Erdler, Margaret R Frey, et al.
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