Latest Conference Coverage

Respiratory Patterns Differ in Pediatric Duchenne Muscular Dystrophy

A recent study presented at MDA 2024 highlighted the evolving respiratory patterns in pediatric patients with Duchenne muscular dystrophy, offering crucial insights for effective respiratory management in this patient population.

US National Registry Analysis Reveals Gender Disparities in Pediatric-Onset Facioscapulohumeral Muscular Dystrophy

A recent analysis of data from the US National Registry presented at MDA 2024 revealed gender disparities in pediatric-onset facioscapulohumeral muscular dystrophy, with girls experiencing more severe outcomes.

Use of the ActiMyo Sensor to Assess 95th Centile of Stride Velocity: Laurent Servais, MD, PhD

The professor of pediatric neuromuscular diseases at the University of Oxford provided insight on a study presented at MDA 2024 assessing the use of a magneto-inertial sensor in ambulant children with Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]

A Case Series Shows Long-Term Efficacy and Convenience of Ravulizumab in Generalized Myasthenia Gravis

A case series presented at the MDA 2024 revealed that ravulizumab infusion intervals show promise in stabilizing symptoms and reducing exacerbations in patients with generalized myasthenia gravis.

Integrating a Business Methodology to Improving Care for ALS: Brooke Eby

At MDA 2024, Brooke Eby shared her perspective as a patient with ALS and the ways to incorporate business approaches to improving clinical care and increasing enrollment for drug trials. [WATCH TIME: 5 minutes]

Preoperative Disease-Modifying Agents Reduce Postoperative Complications in SMA Scoliosis Surgery

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.

Mechanism of Action of Investigational Agent NMD670 for SMA: Jorge Quiroz, MD, MBA

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]

Viltolarsen Shows Significant Pulmonary Function Gains in Ambulant and Nonambulant DMD

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.

Meta Analysis Shows Slow Decline of Muscle Function With Ataluren for Nonsense Mutation DMD

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.

Post Hoc Analysis Shows Consistent Efficacy With Givinostat for DMD

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.

Exon-Skipping Therapy Golodirsen Prolongs Ambulation in DMD, Long-Term Study Shows

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

Vatiquinone Treatment Shows Benefit Across End Points for Friedreich Ataxia in Phase 3 MOVE-FA Trial

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.

Redefining Relapse in Efficacy Measures for Multiple Sclerosis Treatments: Stephen Krieger, MD; Enrique Alvarez, MD, PhD

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]

SRP-9001 Improves Duchenne Muscular Dystrophy Disease Trajectory Despite Failing to Meet Primary End Point in Phase 3 EMBARK Trial

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.

The Intricate Relationship Between Biological Aging and Multiple Sclerosis: Yinan Zhang, MD

The assistant professor of neurology at The Ohio State University Wexner Medical Center talked about results from an ongoing study assessing biological aging in patients with multiple sclerosis using epigenetic clocks and p16INK4a. [WATCH TIME: 6 minutes]

Duchenne Therapy Eteplirsen Safe in Infants as Young as 6 Months, Study Shows

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.

Interim Real-World Data From Phase 4 EVOLVE Study Demonstrate Sustained Treatment Effect of Eteplirsen

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

Tadalafil Improves Microvascular Function in Duchenne Leg Muscles, Study Suggests

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

Safety Profile of Fenebrutinib in MS Similar to Other Autoimmune Conditions

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

Reaction to 2024 MDA Legacy Award: Jeff Chamberlain, PhD

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.

Analyzing Phase 2 CALLIPER Trial of IMU-838 for Progressive MS: Robert J. Fox, MD

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]

Increased Aerobic Glycolysis in Normal Appearing White Matter Revealed as Metabolic Abnormality in Multiple Sclerosis

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.

Clemastine Arm of TRAP-MS Trial Halted Following Increased Disability Accumulation in Progressive 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.

Study Reveals Expanded CD8+ T Cell Clonotypes in Cerebrospinal Fluid in Patients With 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.

Paramagnetic Rim Lesions Predicts Future Cognitive Decline in 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.

Epigenetic Age Acceleration Shows Association With Secondary Progressive 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.

Individual and Group Level Cognitive Benefits of Cladribine Displayed in MAGNIFY-MS Study

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.

Glatiramer Acetate Depot Demonstrates Sustained Long-Term Safety Profile as Potential MS Therapy

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

Ozanimod's Consistent Efficacy in Long-Term Treatment for Multiple Sclerosis: Jeffrey Cohen, MD

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]

Preclinical Data Shows NLRX1 Protects Against Neurodegeneration in Multiple Sclerosis

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.