Latest Conference Coverage

The Potential of Genetic Therapies in Neuromuscular Diseases: Stephan Züchner, MD, PhD

At the 2023 MDA conference, the professor for human genetics and neurology at the University of Miami Miller School of Medicine talked about the role of genetics in neuromuscular diseases and potential therapies. [WATCH TIME: 5 minutes]

Lessons Learned and Future Possibilities in ALS Collaborations: Edritz Javelosa, PhD

At the 2023 MDA conference, the scientific research portfolio director at Muscular Dystrophy Association detailed a session on collaborative research in ALS and ongoing efforts in the field. [WATCH TIME: 3 minutes]

Focus on Gene Therapy and Neuroinflammation in ALS Research: Stanley H. Appel, MD

The director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics at Houston Methodist Hospital spoke on about advances in ALS research and promising therapies on the horizon at the 2023 MDA conference. [WATCH TIME: 8 minutes]

Holistic Approach to Medicine and Rehabilitation in Physical Therapy: Tina Duong, PhD, PT

The director of clinical outcomes and research development in neurology at Stanford University spoke about the paradigm shift in neuromuscular therapy interventions at the 2023 MDA conference. [WATCH TIME: 5 minutes]

Advances in Gene Therapy for Neuromuscular Diseases: Kevin Flanigan, MD

The director of the center for gene therapy at Nationwide Children's Hospital talked about the challenges and opportunities of gene therapies for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]

The Implementation of Platform Trials for ALS Research: Melanie Quintana, PhD

The senior statistical scientist and director of consulting at Berry Consultants talked about innovative clinical trial design in ALS research at the 2023 MDA conference. [WATCH TIME: 5 minutes]

Overcoming the Challenges of Ultra Rare Diseases, Limb Girdle Muscular Dystrophy: Peter Kang, MD

At the 2023 MDA conference, the professor of neurology at the University of Minnesota talked about the current understanding and challenges of Limb Girdle muscular dystrophy from a clinical perspective. [WATCH TIME: 5 minutes]

Importance of Coordination in ALS Collaborative Research: Kuldip Dave, PhD

The senior vice president of research at The ALS Association talked about the complexities of ALS and the importance of collaboration in research at the 2023 MDA conference. [WATCH TIME: 5 minutes]

Understanding the Risk of Multiple Sclerosis in Patients With HIV

Elaine Kingwell, PhD, senior research associate at University College London provided perspective on a study presented at the 2023 ACTRIMS Forum on HIV and multiple sclerosis.

Milestone-Free Time Longer in IV Edaravone-Treated Patients With ALS, Administrative Claims Suggest

Time to milestones such as invasive and noninvasive ventilation, speech-generating devices, and hospice care took significantly longer while on intravenous edaravone.

Maximizing Exercise as Movement Medicine for Neuromuscular Diseases: Donovan Lott, PT, PhD, CSCS

The research associate professor in the department of physical therapy at the University of Florida talked about physical therapy inventions for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]

Standardizing Clinical Practice for End of Life Care in Neuromuscular Diseases: Lauren Treat, MD

The child neurologist at the University of Colorado School of Medicine talked about her presentation on end of life care for patients with neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 5 minutes]

Recapping Research From 2023 MDA Clinical and Scientific Conference

As a recap from MDA 2023, get caught up on some of the latest news in neuromuscular diseases as the NeurologyLive® team shares some of our data updates.

Clinical Trial Experiences Managing Adverse Events in Gene Therapy for DMD: Natalie L. Goedeker, CPNP

At the 2023 MDA Conference, a neurology nurse practitioner in the Neuromuscular Division at Washington University in St. Louis talked about adverse event management of gene therapies in Duchenne muscular dystrophy. [WATCH TIME: 2 minutes]

New MDA Program for Gene Therapy Development in Ultra-Rare Diseases: Sharon Hesterlee, PhD

At the 2023 MDA Conference, the executive vice president and chief research officer of MDA talked about a new program from the MDA to tackle ultra-rare neuromuscular diseases. [WATCH TIME: 5 minutes]

Losmapimod Demonstrates Significant Slowing of Facioscapulohumeral Muscular Dystrophy Over Long-Term Period

Durability of treatment response on reachable workspace was observed among those who continued losmapimod throughout the 96-week period and those who switched from placebo.

Improving Quality of Life, Digestive Health in Duchenne Muscular Dystrophy: David Brumbaugh, MD

The associate professor of pediatrics at the University of Colorado School of Medicine talked at the 2023 MDA Conference about how digestive health impacts patients with Duchene Muscular Dystrophy. [WATCH TIME: 4 minutes]

Myasthenia Agent Zilucoplan Continues to Show Positive Efficacy in Long-Term Extension

In participants who received placebo during the parent studies, rapid improvements were observed as early as week 1 after switching to zilucoplan, and continued through week 12 of the extension period.

Efforts to Expand Neuropalliative Care for Neuromuscular Disorders: Ambereen Mehta, MD, MPH

The palliative care physician and assistant professor of medicine at Johns Hopkins Bayview Medical Center discussed the types of resources clinicians can pull from to expand neuropalliative care for patients with neuromuscular disorders. [WATCH TIME: 3 minutes]

Treg Suppressing Agent COYA 302 Shows Slowing of ALS Disease Progression

After results indicated significant slowing of ALS disease progression, Coya noted it plans to file an IND with the FDA in the second half of 2023 for a potential clinical study thereafter.

NeuroVoices: Emma Ciafaloni, MD, on Mounting Progress in the Neuromuscular Field, Duchenne Muscular Dystrophy

The professor of neurology and pediatrics at University of Rochester Medical Center shared her thoughts on the 2023 MDA Clinical and Scientific Conference, and the progress made in the treatment of neuromuscular disorders.

Raised Awareness and Need For Improvements to End of Life Care in Neuromuscular Disorders: Ambereen Mehta, MD, MPH

The palliative care physician and assistant professor of medicine at Johns Hopkins Bayview Medical Center provided perspective on current state of end-of-life care for neuromuscular disorders and areas to improve upon. [WATCH TIME: 4 minutes]

The Use of Molecular Testing in Limb Girdle Muscular Dystrophy: Douglas Sproule, MD, MSc

The chief medical officer at ML Bio Solutions spoke about the advances in neuromuscular diseases such as limb girdle muscular dystrophy at the 2023 MDA Conference. [WATCH TIME: 3 minutes]

Post-Hoc Analysis of ADAPT Reveals Several Subgroup Responders to Efgartigimod

Efgartigimod-treated patients consistently outperformed placebo regardless of concomitant medication, thymectomy status, and timing of diagnosis.

Post-Hoc Analysis of CHAMPION MG Study Highlights Ravulizumab’s Effects on Specific Muscle Domains

After previously showing positive effects on MG-ADL and QMG, ravulizumab continued to show improvements in ocular and respiratory muscle domains in myasthenia gravis.

Turning Gene Therapy Into Reality: Peter Marks, MD, PhD

The director of the Center for Biologics Evaluation Research at the FDA provided an overview of the Keynote Address he gave at the 2023 Muscular Dystrophy Association Clinical and Scientific Conference. [WATCH TIME: 3 minutes]

Supplementing N-163 Beta Glucan Shows Disease-Modifying Effects as Adjuvant in Duchenne Muscular Dystrophy

More than 90% of patients, ambulatory and non-ambulatory, experienced improvements in Medical Research Council score within 6 months of treatment.

Minor Differences in Inclusion Criteria, Baseline Characteristics Impact Motor and Nonmotor Clinical Outcomes in Presymptomatic Spinal Muscular Atrophy

Differences in baseline characteristics and eligible criteria in presymptomatic infants with spinal muscular atrophy led to differential results on motor and nonmotor clinical outcomes.

Balancing Efficacy and Safety With MS Disease-Modifying Therapies: Omar Al-Louzi, MD

The director of the Visual Outcomes Laboratory at Cedars Sinai talked about the current treatments used in MS and the risks associated with them at 2023 ACTRIMS Forum. [WATCH TIME: 3 minutes]

Using MRIs to Detect Early Signs of Viral Infections in Multiple Sclerosis

At the 2023 ACTRIMS forum, Omar Al-Louzi, MD, director of the Visual Outcomes Laboratory at Cedars Sinai, talked about investigating the relationship between viral infections and multiple sclerosis using MRIs.