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Overall, subcutaneous efgartigimod was safe and effective in patients with seropositive MG, demonstrated by consistent improvements in Myasthenia Gravis-Activites of Daily Living.

Subcutaneous Efgartigimod Demonstrates Efficacy, Safety in Seronegative Myasthenia Gravis

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The trial, a 20-week study assessing lamotrigine in DLB, will use change in Clinical Dementia Rating-Sum of Boxes as the primary efficacy end point, with several other notable secondary outcomes.

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Dean and chief academic officer at the University of Miami Miller School of Medicine.

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Here's the latest multidisciplinary multiple sclerosis (MS) research from the International Journal of MS Care.

Research from IJMSC September 2024

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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

In previously reported data, AOC 1020 demonstrated a consistent reduction in double homeobox 4 regulated genes among patients with facioscapulohumeral muscular dystrophy at 4 months.  

Avidity Biosciences Initiates Biomarker Cohort in Phase 1/2 FORTITUDE Trial, Seeks Accelerated Approval Path for AOC 1020

The NDA includes data from a global placebo-controlled, 72-week study as well as findings from the STRIDE registry, an ongoing, observational, real-world study of ataluren in routine care.

FDA Accepts Resubmitted NDA for Ataluren in Nonsense Duchenne Muscular Dystrophy

Ongoing analysis of vamorolone's effects as a mineralocorticoid receptor antagonist in healthy adults is underway, with results to be presented at upcoming medical conferences.

Vamorolone Shows Potential Dual Mechanism as Mineralcorticoid Receptor Antagonist in LIONHEART Study

Treatment with DYNE-251 revealed dose dependent exon skipping and dystrophin expression as well as improvement in multiple functional end points in both cohorts of the phase 1/2 DELIVER trial.

DYNE-251 Increases Dystrophin Level Expression in Phase 1/2 DELIVER Trial of Duchenne

Screening for DMD in Massachusetts is expected to begin by June 2026, which could enable earlier diagnosis and intervention.

Massachusetts Becomes Fourth State to Include Duchenne Muscular Dystrophy in Newborn Screening Panel

Debra Miller discussed efforts to bring specialized physical therapy and infrastructure support to underserved regions to improve care for patients impacted by Duchenne worldwide

Helping to Expand Global Care for the Duchenne Muscular Dystrophy Patient Community: Debra Miller

"We're very hopeful that by going to underserved countries and communities, we're going to be able to give them care, but [also] give them hope."

Since there is currently no cure for Duchenne muscular dystrophy (DMD), a severe and progressive neuromuscular disease, having comprehensive multidisciplinary care will be important for patients as prior recent studies have shown that this approach has improved life expectancy. To celebrate World Duchenne Day, held September 7, 2024, CureDuchenne launched new global initiatives in efforts to continue to increase awareness of disease that has impacted an estimated 300,000 patients worldwide.

CureDuchenne’s International Outreach Program provides informative training, awareness, and resources to healthcare providers, helping to address the gaps in diagnosing and treating Duchenne to enhance patients' quality of life. After assessing underserved areas for access to education, healthcare, and medical equipment, the organization hosts global events which offer high-level educational programs for clinics and allied health professionals. The program has trained professionals in countries like India, Nepal, Canada, Italy, Russia, England, South Africa, Hungary, Australia, and Ukraine.

, the chief executive officer and founder of CureDuchenne (which was started 21 years ago after her son was diagnosed with DMD), sat down with 

 in an interview to discuss the potential impact of the newly launched global initiatives on improving care standards for DMD. She also shared some insight on the challenges faced in different regions globally when it comes to diagnosing and treating Duchenne, and how CureDuchenne’s recent efforts are helping to address these. Furthermore, Miller spoke about the role of social media and community engagement in the fight for a cure in DMD, especially for World Duchenne Day.

REFERENCES
1. CureDuchenne Expands Global Initiatives and Spreads Awareness for World Duchenne Day 2024. News Release. CureDuchenne. Published August 20, 2024. Accessed September 6, 2024. https://cureduchenne.org/press-release/cureduchenne-expands-global-initiatives-and-spreads-awareness-for-world-duchenne-day-2024/

The chief executive officer and founder at CureDuchenne discussed efforts to bring specialized physical therapy and infrastructure support to underserved regions to improve care for patients impacted by Duchenne worldwide. [WATCH TIME: 5 minutes]

The duo from CureDuchenne talked about a recent webinar that discussed the broad FDA approval of a gene therapy for Duchenne Muscular Dystrophy

Broad FDA Approval of Gene Therapy Expands Treatment Choices for Duchenne Muscular Dystrophy: Debra Miller & Michael Kelly, PhD

"Our job today is far from done in this disease, and we've improved the whole therapeutic landscape of exon skipping. We need to do exactly the same with gene therapy."

Originally SRP-9001 (Elevidys; Sarepta Therapeutics), otherwise known as delandistrogene moxeparvovec-rokl, received FDA approval under the accelerated approval pathway for patients with Duchenne muscular dystrophy (DMD) in June 2023. More recently, the agency granted the AAV vector-based gene therapy 

 for ambulatory patients with DMD. The company announced that this agency decision also includes an accelerated approval for nonambulatory patients, continued approval for which may be contingent upon verification of clinical benefit in a confirmatory trial.

Recently, CureDuchenne hosted webinars in English and Spanish for the Duchenne community after the FDA approved the expansion to the label indication for SRP-9001 to include individuals with DMD with a confirmed mutation in the DMD gene who are at least 4 years of age. The webinars were moderated by Michael Kelly, PhD, the chief scientific officer at CureDuchenne, with 

, a neuromuscular specialist at the Neurology & Neuromuscular Care Center, who shared the latest information and her perspective as a clinician treating patients with this gene therapy.

Following the webinars, Debra Miller, the CEO and founder of CureDuchenne (which was started 21 years ago after her son was diagnosed with DMD), sat down with 

 in an interview to discuss the main topics covered during the webinar on the new gene therapy for DMD. Miller and Kelly also spoke about how the newly approved gene therapy differs from previous treatments for this disease. Moreover, the duo talked about the anticipated future developments in gene therapy and exon skipping for DMD.

REFERENCES
1. Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above. News Release. Sarepta Therapeutics. Published June 20, 2024. Accessed June 20, 2024. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-expanded-us-fda-approval-elevidys
2. Global Nonprofit CureDuchenne to Host Informational Webinars Following FDA Label Expansion of Sarepta Therapeutics’ Gene Therapy, ELEVIDYS. News Release. CureDuchenne. Published June 21, 2024. Accessed July 3, 2024. https://cureduchenne.org/general/global-nonprofit-cureduchenne-to-host-informational-webinars-following-fda-label-expansion-of-sarepta-therapeutics-gene-therapy-elevidys/

The chief executive officer and the chief scientific officer at CureDuchenne talked about a recent webinar that discussed the broad FDA approval of a gene therapy for Duchenne muscular dystrophy, highlighting patient choice and the ongoing efforts to improve treatments. [WATCH TIME: 9 minutes]

Marcello Paglione, PhD, expressed his confidence in givinostat to mitigate the progression of DMD, emphasizing its multifactorial mechanism of action.

Givinostat's Multifactorial Mechanism of Action in Duchenne Muscular Dystrophy: Marcello Paglione, PhD

“We think these are solid data, strong evidence. We are excited to work with the community here to exchange the results and to have a solid scientific conversation with everybody in the community here.

Givinostat (Duvyzat; Italfarmaco SpA), a proprietary histone deacetylase (HDAC) inhibitor, was recently 

 as a treatment for patients with Duchenne muscular dystrophy (DMD), becoming the first nonsteroidal drug approved for all genetic variants of DMD.

The therapy, designed to inhibit HDACs, enzymes prevent gene translation by changing the 3-dimensional folding of DNA in the cell. The data from the phase 3 EPIDYS trial (NCT02851797) assessing givinostat in patients with DMD was the supporting basis of the approval.

EPIDYS, a randomized, double-blind, placebo-controlled, multicenter study, included 179 ambulant male individuals who were randomly assigned 2:1 to either oral givinostat or placebo for an 18-month treatment period. Of these, 120 boys formed the target population. In a new 

 from EPIDYS, findings showed a positive significant difference with givinostat in motor function among patients with DMD in comparison with controls.

 In the overall intention-to-treat (ITT) population (n = 179), investigators reported that givinostat displayed a statistically significant difference compared with controls in change from baseline at 18 months in the timed 4 stair climb (global longitudinal strain mean ratio, 0.84[SD, 0.069] 

The findings from the post hoc analysis, presented at the 

2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

, held March 3-6, in Orlando, Florida, emphasize the efficacy of givinostat in a broader context. At the conference, 

, executive medical lead at Italfarmaco SpA, sat down with 

 to further discuss the role of HDAC in patients with DMD. He also talked about how givinostat's mechanism of action aims to mitigate the progression of the disease. In addition, Paglione spoke about ongoing research initiatives that are being pursued to further understand givinostat's efficacy and safety profile.

The executive medical lead at Italfarmaco SpA expressed his confidence in givinostat to mitigate the progression of Duchenne muscular dystrophy, emphasizing its multifactorial mechanism of action. [WATCH TIME: 5 minutes]

Muscular Dystrophy

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