Muscular Dystrophy Association | Strategic Alliance Partners

The pediatric neurologist at Children’s Hospital of Philadelphia provided background on a notable track session at the upcoming MDA Clinical & Scientific Conference covering updates in gene therapy for neuromuscular conditions. [WATCH TIME: 4 minutes]

The associate professor of neurology at Columbia University gave clinical insight on what neurologists and other neuromuscular specialists can expect from the ALS/frontotemporal dementia track at this year’s MDA Clinical & Scientific Conference. [WATCH TIME: 3 minutes]

Matthew B. Harms, MD, an associate professor of neurology at Columbia University Irving Medical Center, an MDA Care Center, gave clinical insights on the advances in ALS genetic research and what clinicians can expect from the ALS/FTD track at the 2025 MDA Clinical & Scientific Conference.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota talked about the potential of investigating inducted pluripotent stem cells in neuromuscular diseases at MDA’s 2025 conference. [WATCH TIME: 8 minutes]

The clinical researcher at St. Jude Children's Research Hospital discussed the evolving treatment landscape for Charcot-Marie-Tooth disease, which will be featured in a session at the 2025 MDA Conference. [WATCH TIME: 7 minutes]

The chief medical advisor at the Muscular Dystrophy Association shared key highlights of what attendees can expect at the 2025 MDA Clinical and Scientific Conference. [WATCH TIME: 4 minutes]

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.

Donald S. Wood, PhD, president and chief executive officer at the MDA, provided thoughts and perspectives on the promising design and educational attainment for the 2025 MDA Clinical & Scientific conference.

Prior to the 2025 MDA Conference, the vice president of Public Policy & Advocacy at the Muscular Dystrophy Association talked about the increasing interest in gene therapy in the field of neuromuscular diseases.

As part of our monthly clinician spotlight, NeurologyLive® highlighted neuromuscular-associated cardiomyopathy expert Forum Kamdar, MD, PhD, FACC, FHFSA, an advanced heart failure physician-scientist at the University of Minnesota.

The chief research officer of the Muscular Dystrophy Association talked about what is to be expected at the 2025 MDA Conference, held March 16-19, in Dallas, Texas. [WATCH TIME: 5 minutes]

The research grant aims to diversify Friedreich ataxia treatments, addressing challenges in trial design and therapeutic strategies for this condition.

The executive vice president and chief research officer of the Muscular Dystrophy Association shared her reaction to the recent FDA approval of givinostat for Duchenne muscular dystrophy.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Sharon Hesterlee, PhD. [LISTEN TIME: 10 minutes]

The second-year PhD student in bioinformatics at Boston University who lives with LAMA2 congenital muscular dystrophy talked about the potential impact and challenges of gene therapy in neuromuscular diseases. [WATCH TIME: 5 minutes]

Findings from the recent phase 3b SMART trial affirm the safety and efficacy of intravenous onasemnogene abeparvovec (Zolgensma; Novartis) in spinal muscular atrophy when patient weights range from 8.5 kg to 21 kg.

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.

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.

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.

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.

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.

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.

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.