Angela Genge, MD, FRCPC, eMBA, on Targeting STMN2 to Rescue Neurons in ALS
The chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.
Meaningful Activity Improvements Seen in SMA After Nusinersen
Individual items on RULM revealed meaningful improvements in participants from the SHINE and CHERISH studies.
Emma Ciafaloni, MD, on the State of Gene Therapy in Neuromuscular Disease
The professor of neurology and pediatrics at University of Rochester Medical Center spoke about her session at MDA’s 2023 conference.
Giant Axonal Neuropathy Gene Therapy Well-Tolerated With Some Clinical Benefit
New data from a single-site, first-in-human study were presented at the 2023 MDA Conference.
Peter Marks, MD, PhD, on Bringing More Gene Therapies to Patients
The director of the Center for Biologics Evaluation and Research at the FDA discussed his keynote speech at the 2023 MDA Conference.
Nusinersen Well-Tolerated After Zolgensma in SMA
Data from the phase 4 RESPOND trial were presented at the 2023 MDA Conference.
ALS Gene Therapy to be Discussed at the 2023 Muscular Dystrophy Association Clinical & Scientific Conference
Matthew B. Harms, MD, associate professor of neurology at Columbia University, and medical consultant and care center director at the MDA, discussed the session he will be chairing at the conference.
Matthew B. Harms, MD, on Gene Therapy in ALS, MDA 2023
The associate professor of neurology at Columbia University spoke about the session he will be chairing at MDA’s 2023 conference.
Zolgensma Shows Efficacy in SMA With 3 SMN2 Copies
Shephard Mpofu, MD, part of the Novartis Gene Therapies leadership, shared his thoughts on the latest phase 3 data on onasemnogene abeparvovec (Zolgensma) from the SPR1NT trial.
2-year Data for Duchenne Muscular Dystrophy Gene Therapy Shows Durable Improvements
Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.
EMBARK Study Evaluates DMD Gene Therapy
Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.
Presymptomatic SMA Treatment With Zolgensma Associated With Normal Development
New data presented at MDA 2022 showcased positive motor and bulbar function data.
SGT-001 Shows Promising Functional Improvements in DMD
SGT-001 elicited promising improvements in functional and biomarker end points for patients with Duchenne muscular dystrophy.
SRP-9003 Efficacy, Tolerability Sustained at 2 Years in LGMD2E
SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores at 2 years in patients with limb-girdle muscular dystrophy Type 2E.
New Data Showcase Onasemnogene Abeparvovec Efficacy in SMA
Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.
