Improved Respiratory Outcomes for X-Linked Myotubular Myopathy (XLMTM) with Gene Replacement Therapy, Resamirigene Bilparvovec (ASPIRO): Preliminary Results from ASPIRO, a Phase 1/2/3 Study
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE(2022)
Key words
gene replacement therapy,aspiro,improved respiratory outcomes,x-linked
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