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Publications by Thomas A Macek

066 Avxs-101 Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update

Journal of Neurology, Neurosurgery and Psychiatry

English

Related publications

014 AVXS-101 Gene-Replacement Therapy (GRT) in Presymptomatic Spinal Muscular Atrophy (SMA): Study Update

Journal of Neurology, Neurosurgery and Psychiatry

English

P.065 AVXS-101 Gene-Replacement Therapy (GRT)) in Presymptomatic Spinal Muscular Atrophy (SMA): Study Update

Canadian Journal of Neurological Sciences

English

Psy16 - Health Outcome Improvements in Spinal Muscular Atrophy Type 1 Patients With Avxs-101 Gene Replacement Therapy

Value in Health

Occupational Health

English

Psy18 - Rapid Improvements in Motor Function in Spinal Muscular Atrophy Type 1 Following Avxs-101 Gene Replacement Therapy

Value in Health

Occupational Health

English

Spinal Muscular Atrophy Type 1

Pediatric Critical Care Medicine

Intensive Care Medicine

English

P.060 Time to Treatment Effect in Spinal Muscular Atrophy Type 1 (SMA1): An Indirect Comparison of Treatments

Canadian Journal of Neurological Sciences

English

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study With a Prospective Natural History Cohort

Journal of Neuromuscular Diseases

English

Gene Therapy in Mouse Models of Spinal Muscular Atrophy :||Using SMN Gene Replacement to Address Biological Questions

English

Respiratory Management of the Infant With Type 1 Spinal Muscular Atrophy

Archives of Disease in Childhood

English