Muscular Dystrophy Association Clinical and Scientific Conference (MDA)

Data from the HOPE-2-OLE were presented in a late-breaking session at the 2023 MDA Conference.

All children included in the analysis achieved swallowing, oral nutrition, and airway protection outcomes.

Presented at MDA 2023, the gene therapy (also known as delandistrogene moxeparvovec) showed significant improvements on NSAA total score, 10-meter walk/run scores, and time to rise scores relative to an external control cohort.

The chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.

Individual items on RULM revealed meaningful improvements in participants from the SHINE and CHERISH studies.

The professor of neurology and pediatrics at University of Rochester Medical Center spoke about her session at MDA’s 2023 conference.

New data from a single-site, first-in-human study were presented at the 2023 MDA Conference.

The director of the Center for Biologics Evaluation and Research at the FDA discussed his keynote speech at the 2023 MDA Conference.

Data from the phase 4 RESPOND trial were presented at the 2023 MDA 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.

The associate professor of neurology at Columbia University spoke about the session he will be chairing at MDA’s 2023 conference.

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.

Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.

Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.

New data presented at MDA 2022 showcased positive motor and bulbar function data.

SGT-001 elicited promising improvements in functional and biomarker end points for patients with Duchenne muscular dystrophy.

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.

Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.