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Articles

Presented at the 2025 AAN Annual Meeting, findings from a phase 3 study showed that a single 30 mg/kg dose of ANX005 significantly improved health outcomes in Guillain-Barré syndrome, with benefits seen as early as week 1 and sustained over 6 months.

Complement-Targeting Agent ANX005 Demonstrates Therapeutic Efficacy in Phase 3 Study of Guillain-Barré Syndrome

Findings showed that atogepant 60 mg once daily demonstrated significant improvements in functional outcomes for patients with episodic migraine, compared with placebo, over 12 weeks.

Post Hoc Analysis of ADVANCE Trial Reveals Atogepant Improves Functional Outcomes in Migraine 

Long-term data from a phase 2 study extension suggest that frexalimab, a CD40L inhibitor, maintains disease control and is well-tolerated in relapsing multiple sclerosis, reinforcing its potential as a next-generation treatment.

Two-Year Open-Label Data Highlight Frexalimab’s Impact on Disease Activity in Relapsing Multiple Sclerosis

A recent post hoc analysis of the REST-ON trial reported that once-nightly sodium oxybate did not worsen the apnea-hypopnea index in individuals with narcolepsy with no or mild sleep apnea.

Post Hoc Analysis Shows Once-Nightly Sodium Oxybate Does Not Worsen Apnea-Hypopnea Index in Narcolepsy

Senior vice president of research at The ALS Association. 

Videos

The senior vice president of research at The ALS Association talked about a multidisciplinary session presented at the 2025 MDA Conference that highlighted diverse strategies for neural repair, from pharmacological targets to noninvasive brain stimulation. [WATCH TIME: 6 minutes]

Exploring Multimodal Approaches to Neural Regeneration: Kuldip Dave, PhD

Marco Meglio, Assistant Managing Editor for 

, has been with the team since October 2019. Follow him on Twitter 

In this final episode, neurologists discuss the role of advanced imaging, like OCT, in diagnosing and managing MS, emphasizing the need for further research to optimize its clinical application. [WATCH TIME: 2 minutes]

Bridging Neurology and Ophthalmology in Multiple Sclerosis Research

A pair of neurologists provide clinical insight on the difficulties with identifying and differentiating optic neuritis in various autoimmune conditions like multiple sclerosis and neuromyelitis optica spectrum disorder. [WATCH TIME: 4 minutes]

Challenges in Diagnosing Optic Neuritis in MS and MOGAD

In this third episode, experts delve into the evolving role of optic neuritis and the central vein sign in refining multiple sclerosis diagnosis, highlighting their significance in distinguishing MS from other conditions and ensuring accurate patient care. [WATCH TIME: 3 minutes]

Educating the Clinical Community on Changes to MS Diagnostic Criteria

NeurologyLive is your direct connection to the latest neurology news and interviews with expert neurologists in multimedia formats.

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1.125rem

Clinical

 Muscular Dystrophy Disease Spotlight page offers specific coverage on the latest expert conversations and clinical trial data associated with the treatment and management of patients with muscular dystrophies.

 to subscribe on your favorite streaming service.

 podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis (MS), Parkinson disease, dementia, sleep disorders, and more.

This episode, "FDA Approves HDAC Inhibitor Givinostat for Duchenne Muscular Dystrophy," features an exclusive interview with 

, executive vice president and chief research officer of the 

, who sat down to discuss the significance of the FDA approval of givinostat (Duvyzat; Italfarmaco) and how it changes the care for patients with DMD. She spoke specifically about the mechanism of action of the therapy as a proprietary histone deacetylase (HDAC) inhibitor, its safety profile, and how it may be used with other agents. In addition, she discussed other related topics on gene therapy and unmet needs for this patient population.

For more of NeurologyLive's coverage of givinostat's approval, head here: 

FDA Approves Italfarmaco's Givinostat for Duchenne Muscular Dystrophy

 – Givinostat approved for Duchenne muscular dystrophy

 – Sharon Hesterlee, PhD, on the approval's implications

 – Positive downstream effects of the approval

 – Hersterlee on the safety profile of givinostat

 – Overcoming roadblocks involved with gene therapy 

 – Remaining unmet needs for patients with Duchenne

 podcast. Be sure to leave a rating and review for the show. Thanks for listening!

REFERENCES
1. FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy. FDA. News release. March 21, 2024. Accessed March 29, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy
2. Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. 

. 2024;23(4):393-403. doi:10.1016/S1474-4422(24)00036-X

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

Special Episode: FDA Approves HDAC Inhibitor Givinostat for Duchenne Muscular Dystrophy

This episode, "FDA Green-Lights Vamorolone for Duchenne Muscular Dystrophy," features an exclusive interview with Barry J.  Byrne, MD, PhD, chief medical advisor, 

, and associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida. He discussed the recent FDA approval of Santhera Pharmaceuticals' investigational agent vamorolone (now marketed as Agamree, and soon to be acquired by Catalyst Pharmaceuticals in North America), its potential to affect the treatment paradigm as a first-in-class dissociative steroid, and important efficacy and safety information from the phase 2b VISION-DMD study (NCT03439670).

Click here to read more of NeurologyLive's coverage of the vamorolone approval. 

 – Vamorolone approved for Duchenne muscular dystrophy

 – Barry J. Byrne, MD, PhD, on his immediate reaction

 – Byrne on the importance of bone mineralization in DMD

 – Phase 2b efficacy data from VISION-DMD

 – Byrne with context on the efficacy data

 – Byrne with context on the landscape of DMD therapeutic development and personalization approaches

REFERENCES
1. Catalyst Pharmaceuticals Reports FDA Approval of AGAMREE® (vamorolone) for Duchenne Muscular Dystrophy Granted to Santhera Pharmaceuticals. News release. Catalyst Pharmaceuticals. October 26, 2023. Accessed October 26, 2023. https://www.globenewswire.com/en/news-release/2023/10/26/2767947/13009/en/Catalyst-Pharmaceuticals-Reports-FDA-Approval-of-AGAMREE-vamorolone-for-Duchenne-Muscular-Dystrophy-Granted-to-Santhera-Pharmaceuticals.html
2. Santhera and ReveraGen announce positive and statistically highly significant topline results with vamorolone in pivotal VISION-DMD study. News release. Santhera. June 1, 2021. Accessed February 1, 2023. https://www.globenewswire.com/news-release/2021/06/01/2239124/0/en/Santhera-and-ReveraGen-Announce-Positive-and-Statistically-Highly-Significant-Topline-Results-with-Vamorolone-in-Pivotal-VISION-DMD-Study.html
3. Dang U, Guglieri M, Clemens PR, et al. Delayed start analysis of efficacy outcomes in placebo to vamorolone crossover participants in VBP15-004. Presented at MDA Clinical and Scientific Conference; March 13-16. Poster 89.
4. Muscular Dystrophy Association-Supported Drug AGAMREE® (vamorolone) Approved for the Treatment of Duchenne Muscular Dystrophy by the FDA. News release. MDA. October 26, 2023. Accessed October 26, 2023. https://www.mda.org/press-releases/mda-supported-drug-agamree-vamorolone-approved-for-the-treatment-of-duchenne-muscular-dystrophy-by-the-fda
5. Guglieri M, Clemens PR, Perlman SJ, et al. Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: a randomized clinical trial. 

Published online August 29, 2022. doi:10.1001/jamaneurol.2022.2480

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Barry J. Byrne, MD, PhD. [LISTEN TIME: 10 minutes]

Special Episode: FDA Green-Lights Vamorolone for Duchenne Muscular Dystrophy

This episode, "SRP-9001 Approved As First Gene Therapy for Duchenne Muscular Dystrophy," features an exclusive interview with Natalie Goedeker, CPNP, a nurse practitioner in neurology in the Neuromuscular Division at Washington University in St Louis. SRP-9001 (Elevidys; Sarepta), also known as delandistrogene moxeparvovec, was approved for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the 

 gene aged 4 to 5 years, based on data from the 3 clinical trials.

 – SRP-9001 (Elevidys; Sarepta) is approved for DMD

 – Natalie Goedeker, CPNP, on the approval

 – Goedeker on pieces of efficacy data released

 – Currently available therapies for DMD

 – Goedeker on role of SRP-9001 in treatment landscape

– Goedeker on safety considerations of which prescribing clinicians should be aware

REFERENCE
1. Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. News release. June 22, 2023. Accessed June 22, 2023. https://www.businesswire.com/news/home/20230622454844/en/

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Natalie Goedeker, CPNP. [LISTEN TIME: 12 minutes]

Special Episode: SRP-9001 Approved As First Gene Therapy for Duchenne Muscular Dystrophy

 podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis, Parkinson disease, dementia, sleep disorders, and more.

Episode 80, "Advancing Treatment for Rare Neuromuscular Disorders," features an exclusive interview with Sarah Boyce, president and CEO of Avidity Biosciences, who shared insight into the company's ongoing work with its antibody oligonucleotide conjugates (AOC) platform and the focus on myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD) treatments, as well as the challenges with developing successful RNA therapies, and more.

This episode is brought to you by the Giants of Multiple Sclerosis®. This premier neuroscience award program celebrates pioneers, innovators, and future generations of leaders for their remarkable achievements in Multiple Sclerosis. 

The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here:

Anti-CD20 Therapy Ublituximab Gains FDA Approval for Relapsing Multiple Sclerosis

FDA Clears First Stroke-Specific Radial Access Platform for Mechanical Thrombectomy

FDA Designates Myasthenia Gravis Agent Rozanolixizumab Application for Priority Review

FDA Approves Eisai’s Lecanemab for the Treatment for Alzheimer Disease

FDA Accepts sBLA for Revance’s Cervical Dystonia Treatment DaxibotulinumtoxinA

 – Current limitations with RNA therapies

 – The decision to focus on rare genetic muscle diseases

– The patient needs in DM1, FSHD, and DMD

 – Other clinical areas of focus and collaboration for Avidity

 – Upcoming data presentations planned for 2023 in DMD and myotonic dystrophy

 – The benefits of collaboration in the rare disease community

 – Looking ahead to the future of therapies in rare muscular disease

Hoping to listen on your favorite podcast app? See below:

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Sarah Boyce. [LISTEN TIME: 21 minutes]

Episode 80: Advancing Treatment for Rare Neuromuscular Disorders

The European Medicines Agency has requested a pause on 3 clinical trials of FDA-approved gene therapy Elevidys following the death of a patient who suffered acute liver failure after treatment.

Sarepta’s Duchenne Gene Therapy Trials Paused in Europe Amid Patient Death Investigation

The HARBOR study is an ongoing phase 3 global study focused on assessing del‑desiran, formerly known as AOC 1001, which aims to treat the underlying cause of DM1.

Evaluating the Therapeutic Potential of Del-Desiran in Myotonic Dystrophy Type 1: The HARBOR Trial

Pooled safety data of delandistrogene moxeparvovec, a gene transfer therapy for Duchenne muscular dystrophy, suggests a manageable tolerability profile up to 5 years of follow-up. 

Duchenne Gene Therapy Delandistrogene Moxeparvovec Shows Manageable Safety Over 5-Year Period

New biomarker data presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference of RGX-202 in the phase 1/2 trial highlight strong microdystrophin expression.

Gene Therapy RGX-202 Shows Positive Interim Results in Duchenne Muscular Dystrophy Trial

A new analysis reported that givinostat, an oral histone deacetylase inhibitor recently approved for DMD, slowed disease progression by approximately 2 years compared with standard care.

Givinostat Delays Duchenne Muscular Dystrophy Progression by 2 Years, Simulation Model Shows

The professor of medicine at the University of Minnesota discussed the progress and challenges of cell therapy in treating muscular dystrophy, highlighting new research on stem cells and the potential for clinical application.

Expanding the Development of Cell Therapy for Muscular Dystrophy: Rita Perlingeiro, PhD

"So, hopefully, you’re fixing 2 things: you’re fixing what’s missing, but also fixing the homeostasis of the muscle with healthy muscle."

 in Dallas, Texas. The annual 4-day event brings together scientific and medical professionals, patients, and advocacy groups to explore the latest advances in the field of neuromuscular disorders.

 The conference will feature presentations on all different types of neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, spinal muscular atrophy, limb-girdle muscular dystrophy, myasthenia gravis, Charcot-Marie-Tooth (CMT) disease, and much more.

In this upcoming meeting, session tracks will feature topics including strategies for regenerating muscle and nerve tissue, new insights into disease mechanisms, clinical experience in gene therapy, access and reimbursement, and clinical trial readouts. At the conference held last year, 1492 in-person and 567 virtual attended from over 30 countries, which made it the biggest global gathering of the neuromuscular disease community. For this year, those who are registered with MDA have the opportunity to partake in the virtual meeting at no-cost or may register in-person at the patient/caregiver rate until allotted spots are filled.

Prior to the event, conference track chair 

, a professor of medicine at the University of Minnesota, sat down with 

 in an interview to discuss key challenges in translating cell therapies from the lab to clinical practice for patients with muscular dystrophy. In the discussion, she explored the differences between pluripotent stem cells and more differentiated cells like myoblasts, highlighting their long-term therapeutic potential. Perlingeiro also addressed the critical role the muscle environment plays in the success of cell therapies and emphasized strategies for optimizing this environment to improve treatment outcomes.

Registration for the 2025 MDA Conference is now open! The meeting is set to be held at the Hilton Anatole, Dallas, Texas, from March 16 to 19, 2025. To register and for more information, head to 

REFERENCES
1. Registration Now Open for the 2025 MDA Clinical & Scientific Conference Featuring the Latest Innovations in Neuromuscular Disease Research and Care with Global Leaders, March 16-19 in Dallas, TX. News Release. MDA. July 30, 2024. Accessed September 25, 2024. https://www.mda.org/press-releases/2024/registration-now-open-for-the-2025-mda-clinical-and-scientific-conference-featuring-the-latest-innovations-in-neuromuscular-disease-research-and-care-with-global-leaders-march-16-19-in-dallas-tx

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]  

SAP Partners

The associate vice president of community outreach at CureDuchenne talked about events like the 2025 FUTURES National Conference that aim to bring individuals from the Duchenne community together.

Engaging the Clinical and Patient DMD Community at the 2025 FUTURES Conference: Heather Medlin, MSW

"We’re really looking to bring everyone together under one roof, in one space, to both focus on community care, answer questions, and, most importantly, give people time to connect."

Throughout the 2025 calendar year, CureDuchenne will host free full-day 

across the country to provide essential resources and highlight the latest advancements in care for patients with Duchenne muscular dystrophy (DMD) and their families. These workshops will feature expert-led discussions on multidisciplinary care, physical therapy techniques, and clinical trial updates. Local healthcare providers and CureDuchenne’s scientific team will engage with families in an interactive setting, offering practical guidance and access to valuable community resources. Each workshop includes an “Open House” with local support organizations, as well as breakfast, lunch, travel assistance for eligible attendees, and translation services to ensure accessibility for all participants.

In addition to these regional events, CureDuchenne will host its annual 

, held May 22-25 at the JW Marriott San Antonio Hill Country Resort & Spa in San Antonio, Texas. This multi-day conference will bring together families, caregivers, clinicians, and researchers to explore groundbreaking treatments, care strategies, and emerging research in DMD and Becker muscular dystrophy. Attendees will have opportunities to engage in expert-led sessions, participate in networking events, and enjoy community-building activities, including a dedicated gaming lounge for children. To foster inclusivity, translation services will be available for all sessions, and a virtual attendance option will be offered.

Building on the insights and resources offered through CureDuchenne’s 2025 events, Heather Medlin, MSW, the associate vice president of community outreach of the organization, recently sat down for an interview with 

 to discuss key issues surrounding Duchenne care. In the interview, Medlin explored how evolving treatment options are reshaping the long-term care needs of individuals with Duchenne. She also addressed the significant barriers to accessing Duchenne care and discussed potential solutions to improve healthcare equity for affected families. Furthermore, Medlin highlighted the importance of integrating culturally and linguistically inclusive tracks to foster better patient and caregiver engagement, ensuring that everyone has the opportunity to fully participate in the Duchenne care community.

REFERENCES
1. CureDuchenne Announces Educational Events for Families and Caregivers of Individuals with Duchenne or Becker Muscular Dystrophy. News Release. Published January 31, 2025. Accessed February 13, 2025. https://cureduchenne.org/all-news/cureduchenne-announces-educational-events-for-families-and-caregivers-of-individuals-with-duchenne-or-becker-muscular-dystrophy-2/

The associate vice president of community outreach at CureDuchenne talked about events like the 2025 FUTURES National Conference that aim to bring individuals from the Duchenne community together. [WATCH TIME: 6 minutes]

The chief medical advisor of the Muscular Dystrophy Association discussed what attendees can expect at the 2025 MDA Meeting.

 Barry J Byrne, MD, PhD, on MDA’s Upcoming 2025 Clinical & Scientific Conference

"his is the era of therapies for neuromuscular diseases. In the past year, we saw an additional few strategies that are transformative in the care of boys with DMD and we have continued to make advances in SMA care following on the enormous success of the release of Zolgensma in 2019."

This content was originally published by our sister publication,

 (MDA) is an organization dedicated to serving the community of patients with neuromuscular disorders, both those with muscular dystrophies and those with other types of neuromuscular disease. Each year, the organization holds the MDA Clinical & Scientific Conference to bring together physicians, clinical trial investigators, academic researchers, patient advocates, biotech executives, and other stakeholders to share and discuss the latest findings in neuromuscular disease research. This year’s meeting, which will be held from March 16 to 19, 2025, in Dallas, Texas, is notable for coinciding with the 75th anniversary of the organization itself.

, the chief medical advisor at MDA, to learn more about what attendees can expect at this year’s event. He emphasized that the field of muscular dystrophy has recently entered the era of disease-modifying treatment after a long history of basic research, thanks in part to the FDA’s approvals of several gene therapy products for neuromuscular diseases in recent years. Furthermore, he noted that means of maximizing muscle regeneration in patients with degenerative neuromuscular diseases, real world experience with Elevidys following its 2024 expanded access approval, and tackling challenges with access to treatment or reimbursement will be among the themes at the 2025 conference.

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]

Muscular Dystrophy

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