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2. Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy.

3. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

5. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

6. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

7. Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians

8. Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy

9. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

10. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

16. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR)

18. Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

21. Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy

22. sj-docx-1-tan-10.1177_17562864221149781 – Supplemental material for Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy

28. Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network

35. OUTCOME RELIABILITY IN NON-AMBULATORY BOYS/MEN WITH DUCHENNE MUSCULAR DYSTROPHY

40. Six Month Results of Spinraza in Adults with Spinal Muscular Atrophy (SAS) Study (2446)

42. The Spinal Muscular Atrophy Health Index: A novel outcome for measuring how a patient feels and functions

46. Medical management of muscle weakness in Duchenne muscular dystrophy

49. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2at risk for spinal muscular atrophy: the Phase III SPR1NT trial

50. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

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