Latest Conference Coverage

Advances in Genetics to Understand Brain Health in Duchenne Muscular Dystrophy: Mathula Thangarajh, MD, PhD

The neuromuscular physician at Virginia Commonwealth University talked about the promising future of gene therapy and access to resources in Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

Assessing New Long-Term Efficacy and Durability Data of Zolgensma for Spinal Muscular Atrophy

Sitra Tauscher-Wisniewski, MD, vice president clinical development and analytics, Novartis Gene Therapies, talked about the new data findings from a study on gene therapy Zolgensma at the 2023 MDA conference.

Predicting Clinical Needs and Potential for Genetic Therapies in ALS: Jennifer Morganroth, MD, MBA

At the 2023 MDA’s Clinical and Scientific conference, the neurology resident at Penn Medicine talked about the challenges of delivering intrathecal therapies and the importance of planning clinical resources for patients with ALS. [WATCH TIME: 3 minutes]

Cerevance Announces Promising Data Updates on Innovative Approaches to Treating Parkinson Disease

Revolutionizing Treatment For Duchenne Muscular Dystrophy with Gene Editing: Courtney Young, PhD

The co-founder and chief executive officer of MyoGene Bio talked about the advantages of gene editing over traditional gene therapy as a long term treatment for neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 3 minutes]

Physical Therapy, Optimizing Function in Neuromuscular Diseases: Megan Iammarino, PT, DPT

The research physical therapist at Nationwide Children’s Hospital talked about the role of physical therapy in the changing landscape of care with neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 5 minutes]

The Development of New Gene Therapies to Address Unmet Needs in ALS: Matthew B. Harms, MD

The associate professor of neurology at Columbia University spoke at the 2023 MDA conference about the unmet needs of patients with ALS and the potential of new gene therapies. [WATCH TIME: 5 minutes]

Early Monitoring and Treatment for Bone Health in Duchenne Muscular Dystrophy: Meilan Rutter, MD

At the 2023 MDA conference, a pediatric endocrinologist from Cincinnati Children’s Hospital talked about the unmet needs in treating bone health for patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

Personalized Treatment Approaches of Myopathies Based on Antibodies: Suur Bilicier, MD

The associate professor of neurology at the University of Texas Health Science Center at Houston talked about advances in the classification of inflammatory myopathies at the recently concluded 2023 MDA conference. [WATCH TIME: 4 minutes]

Top Interviews from 2023 MDA: Expert Insights on Neuromuscular Diseases

A group of experts in the care of patients with neuromuscular diseases—Douglas Sproule, MD, MSc; David Brumbaugh, MD; Sharon Hesterlee, PhD; Lauren Treat, MD; Donovan Lott, PT, PhD, CSCS—shared their perspectives on hot topics of treatment and management from the 2023 MDA conference.

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.