Latest Conference Coverage

ND0612 Significantly Improves ON Time in Parkinson Disease in Phase 3 BouNDless Trial

Compared with immediate-release levodopa/carbidopa, NeuroDerm’s 24-hour subcutaneous liquid infusion improved ON time without dyskinesia and with nontroublesome dyskinesia.

Atogepant Reports Positive Data for Episodic Migraine in Phase 3 Trial

Findings showed a significant reduction in mean monthly migraine days with atogepant 60 mg once daily for patients with episodic migraine compared with placebo across a 12-week period.

Ataluren Confirms Benefit in Nonsense Mutation DMD With Significant Results in Subgroup Analysis

In a subgroup of patients with 300 m to 400 m 6MWD scores in the phase 3 Study 041, ataluren (Translarna; PTC Therapeutics) showed particular improvements from baseline.

Preserving Cognitive Status in Pediatric Multiple Sclerosis: Kimberly O’Neill, MD

The pediatric neuroimmunology fellow at NYU Langone provided perspective on a study presented at AAN 2023 comparing cognitive trajectories of pediatric and adult multiple sclerosis. [WATCH TIME: 3 minutes]

Zavegepant Nasal Spray Proves to Be an Effective Acute Migraine Treatment

Zavegepant, a calcitonin gene-related peptide (CGRP) receptor antagonist, provided onset of pain relief as early as 15 minutes postdose and sustained benefits to 48 hours post dose.

Long-term Ofatumumab Treatment Shows Freedom From Disease Progression, Favors Earlier Initiation

The data, from the phase 3 ASCLEPIOS I and II trials and ALITHIOS open-label extension, suggest that most patients remained free from disease progression, with most progression being independent of relapse activity.

Ocrelizumab Induced Significant Depletion of B-cell Subsets in Relapsing-Remitting MS

An interim analysis showed no reduction in T-cell counts and their subsets as a consequence of B-cell depletion in the first year of treatment with ocrelizumab.

Phase 3 CHAMPION-NMOSD Trial With Ravulizumab Significantly Lowers Risk of Relapse

Updated results to the phase 3 CHAMPION-NMOSD trial of ravulizumab showed significantly lower HAI score worsening following treatment.

Targeting Neuroinflammation in Huntington Disease: Rajeev Kumar, MD

The medical director of the Rocky Mountain Movement Disorders Center talked about the motivations of the phase 2 study on ANX005 for Huntington disease at the 2023 AAN Annual Meeting. [WATCH TIME: 3 minutes]

Eculizumab Safe and Effective in Adolescents With Myasthenia Gravis, Small-Scale Study Suggests

FDA-approved for adults with generalized myasthenia gravis in 2016, eculizumab continued to show promising efficacy and safety responses in adolescents with the disease, regardless of on concomitant use of IVIG.

Identifying Gaps in Access to Accurate Information on Epilepsy Surgery: Daniel Correa, MD, MSc

The neurologist at Montefiore Medical Center, Albert Einstein College of Medicine, provided insight on the need to improve online resources that provide epilepsy surgery information to patients. [WATCH TIME: 4 minutes]

Associations Between Organ Health and Brain Function, Preserving Brain Health: Costantino Iadecola, MD

The director of the Feil Family Brain and Mind Research Institute at Weill Cornell Medicine spoke about preserving brain function through medical, sociopolitical, and educational efforts as part of his presentation at the 2023 AAN annual meeting. [WATCH TIME: 6 minutes]

Extended-Release Carbidopa/Levodopa Shows Tolerable Safety, Maintained Efficacy in Open-Label Extension

The majority of treatment-emergent adverse events from IPX203 were mild or moderate in nature, and occurred within the first 90 days of treatment.

New 12-Month Findings Highlight ALZ-801’s Clinical Effect on Alzheimer Biomarkers, Hippocampal Atrophy

Treatment with ALZ-801, an investigational agent in development for Alzheimer disease, resulted in more than 40% reduction in plasma p-tau181 at the end of the 12-month analysis.

Open-Label Analysis Highlights Long-Term Safety of BTK Inhibitor Tolebrutinib

There were no observed tolebrutinib dose effect for treatment-emergent adverse events in the double-blind portion, as well as no new safety signals seen for those who crossed over in the open-label extension.

Targeting Blood Vessels to Improve Treatment for Alzheimer Disease: Costantino Iadecola, MD

The director of the Feil Family Brain and Mind Research Institute at Weill Cornell Medicine talked about the importance of vascular health in preventing cognitive impairment as part of his presentation at the 2023 AAN annual meeting. [WATCH TIME: 6 minutes]

Efficacy of Rozanolixizumab Seen Across Several Subgroups of Myasthenia Gravis

Rozanolixizumab has shown promising effects as a potential treatment for myasthenia gravis, as explained by high response rates on MG Activities of Daily Living and other measures.

Advances in Neurology, Contributions to Brain Health: Costantino Iadecola, MD

The director of the Feil Family Brain and Mind Research Institute at Weill Cornell Medicine talked about brain health in relation to vascular health ahead of the 2023 AAN annual meeting. [WATCH TIME: 3 minutes]

Restoring Protein Levels With QRL-201 Has Potential to Stabilize Disease Progression in ALS: Angela Genge, MD, FRCPC, eMBA

The chief medical officer of QurAlis talked about the potential of QRL-201 in patients with ALS from her talk presented at the 2023 MDA conference. [WATCH TIME: 4 minutes]

Clinical Application of Induced Pluripotent Stem Cells in Muscular Dystrophy: Rita Perlingeiro, PhD

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 2023 conference. [WATCH TIME: 5 minutes]

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.