Latest Conference Coverage

A recent study presented at MDA 2024 suggests that preoperative use of disease-modifying agents for patients with spinal muscular atrophy leads to less severe postoperative complications following scoliosis surgery.

The chief medical officer of NMD Pharma shed light on a newly announced phase 2 study assessing NMD670, a neuromuscular transmission enhancer, for patients with spinal muscular atrophy. [WATCH TIME: 4 minutes]

In a recent phase 2 trial analysis of viltolarsen presented at MDA 2024, findings showed improvement in forced vital capacity compared with standard care in patients with Duchenne muscular dystrophy.

A new meta analysis of 3 studies presented at MDA 2024 revealed that ataluren significantly slowed the decline in muscle function for patients with nonsense mutation Duchenne muscular dystrophy.

A recent post hoc analysis of the phase 3 EPIDYS trial presented at MDA 2024 revealed significant positive outcomes with givinostat, a histone deacetylase inhibitor, among patients with Duchenne muscular dystrophy.

Median time to loss of ambulation for golodirsen-treated patients was 1968 days vs 1092 days for external control patients.

A new analysis of phase 3 MOVE-FA trial presented at MDA 2024 revealed promising outcomes for vatiquinone, an oral 15-lipoxygenase inhibitor, in patients with Friedreich ataxia.

The associate professor of neurology at Icahn School of Medicine at Mount Sinai and associate professor of neurology at University of Colorado School of Medicine talked about the potential of redefining relapses in the landscape of clinical trials for multiple sclerosis. [WATCH TIME: 3 minutes]

Over a 52-week treatment period, treatment with SRP-9001 resulted in improvements in secondary outcomes of time to rise, micro-dystrophin expression, and 10-meter walk/run.

Patients with Duchenne muscular dystrophy between ages of 6 and 24 months old demonstrated a similar safety profile on eteplirsen than those between 24 and 48 months of age.

In a real-world study, eterplirsen was safe for patients with Duchenne muscular dystrophy, with sustained or improved status in function.

Tadalafil shows potential in addressing microvascular impairment in Duchenne muscular dystrophy, with post-contractile MRI as a screening tool.

Phase 2 study analysis of Roche's fenebrutinib reveals similar safety profiles across multiple autoimmune indications, including relapsing multiple sclerosis.

Jeff Chamberlain, PhD, a leading professor in gene therapy focused on Duchenne muscular dystrophy, shared his reaction to being named the recipient of the 2024 MDA Legacy Award, as well as the state of the DMD field currently.

The staff neurologist at the Mellen Center for Multiple Sclerosis at Cleveland Clinic talked about findings from an interim analysis of the phase 2 CALLIPER trial assessing vidofludimus calcium in progressive multiple sclerosis. [WATCH TIME: 4 minutes]

A recent study presented at the 2024 ACTRIMS Forum suggests that increased aerobic glycolysis in normal appearing white matter may be an early feature of multiple sclerosis.

New data from the TRAP-MS trial showed clemastine fumarate's association with increased disability accumulation in cases of non-lesional multiple sclerosis.

A recent study presented the 2024 ACTRIMS Forum showed that a subset of CD8+ T cells preferentially expanded in the cerebrospinal fluid of patients with multiple sclerosis.

A recent study presented at the 2024 ACTRIMS Forum revealed the association between paramagnetic rim lesions and subsequent cognitive decline in patients with multiple sclerosis.

New findings from a study presented at the 2024 ACTRIMS Forum revealed an increase in epigenetic age acceleration among a subgroup of patients with multiple sclerosis.

Using a 4-point score change on Symbol Digits Modalities Test, more than 80% of patients had either sustained improvement or no sustained change after 2 years of treatment with cladribine.

GA Depot, which remains under review by the FDA, showed a safe and tolerable profile in an additional 52-week extension period.

The director of Cleveland Clinic’s Mellen Center for Multiple Sclerosis Treatment and Research talked about recent findings on long-term impact of ozanimod in patients with relapsing-remitting multiple sclerosis presented at ACTRIMS Forum 2024. [WATCH TIME: 4 minutes]

A new study showed that NLRX1, a mitochondrial innate immune sensor, may serve as a promising therapeutic target to prevent inflammatory neurodegeneration in multiple sclerosis.

The chief medical advisor at the Muscular Dystrophy Association provided perspective on the upcoming meeting and the conversations surrounding new therapeutics for diseases that once had little to nothing. [WATCH TIME: 4 minutes]

A recent study presented at 2024 ACTRIMS Forum showed that body mass index may influence disease progression in patients with multiple sclerosis.

These data, presented at 2024 ACTRIMS Forum, highlight the impacts neurofilament light has on patients with multiple sclerosis irrespective of race or ethnicity.

Long-term data from the phase 3 DAYBREAK trial affirmed sustained efficacy of ozanimod for relapsing forms of multiple sclerosis, with a high amount of patients who were relapse-free at 6 years.