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Publications
647
The 100,000 Genomes Pilot on Rare Disease Diagnosis in Healthcare − A Preliminary Report
Authors:
Damian Smedley
,
Katherine R. Smith
,
Antonio Rueda Martin
…
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
Authors:
Perry B Shieh
,
Nancy L Kuntz
,
James J Dowling
…
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy : A 30-Month Nonrandomized Controlled Open-Label Extension Trial
Authors:
Jean Mah
,
Paula Clemens
,
Michela Guglieri
…
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Francesco Muntoni
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