Lesa Mehl

Safety and efficacy of intravenous onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1: interim analysis from LT-001, a long-term follow-up study of patients from the START study

Megan A. Waldrop, Roberto Bernardo Escudero, Lina Yang, Rebekah A. Camarata et autres

us, cn, ch (code pays fourni par la source)

Systematic literature review of the impact of spinal muscular atrophy therapies on bulbar function

Katlyn McGrattan, Katie Walsh, Lesa Mehl, Simarjeet Kaur et autres

us, in (code pays fourni par la source)

179P Intravenous and intrathecal onasemnogene abeparvovec gene therapy in symptomatic and presymptomatic SMA: long-term follow-up study

B. Darras, Michelle A. Farrar, E. Mercuri, K. Strauss et autres

P223 Long-term follow-up of onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy (SMA) type 1

Jerry R. Mendell, M. Wigderson, I. Alecu, Lu Yang et autres

us, ch (code pays fourni par la source)

P211 Intravenous and intrathecal onasemnogene abeparvovec gene therapy in symptomatic and presymptomatic spinal muscular atrophy (SMA): long-term follow-up study

Basil T. Darras, Eugenio Mercuri, K. Strauss, John Day et autres

us, it, ch (code pays fourni par la source)

Long-Term Follow-Up of Onasemnogene Abeparvovec Gene Therapy in Patients with Spinal Muscular Atrophy Type 1 (P7-9.006)

J. Mendell, Melissa Wigderson, I. Alecu, Lina Yang et autres

us, fr, ch (code pays fourni par la source)

Intravenous and Intrathecal Onasemnogene Abeparvovec Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy: Long-Term Follow-Up Study (S34.002)

Basil T. Darras, Eugenio Mercuri, Kevin A. Strauss, John Day et autres

us, tw, it, fr, ch (code pays fourni par la source)