Latest Conference Coverage

The pediatrician in chief at Johns Hopkins Children’s Center outlined the logistical, ethical, and regulatory hurdles of conducting pediatric MS trials, emphasizing the need for faster, more inclusive study designs to bring emerging therapies to clinic. [WATCH TIME: 5 minutes]

Apitegromab, an invesigational muscle-targeting treatment, showed promising results in improving motor function among young patients with SMA in a phase 2 trial.

At CNS 2025, the child neurologist at Rady Children's Hospital-San Diego talked about the expanding role of genetics for diagnosis and treatment in pediatric neurology. [WATCH TIME: 5 minutes]

In this post-meeting Q&A, Claire Henchcliffe, MD, PhD, president-elect of the AALN, shared her reactions to AALN’s second annual meeting, the conversations that mattered most, and how the group plans to broaden and support neurology leadership in the year ahead.

Johan Luthman, executive vice president and head of R&D at Lundbeck, discussed the rationale behind amlenetug, its mechanism of action, and the company’s ongoing efforts in multiple system atrophy through the phase 3 MASCOT trial.

The professor of neurology at Barts and London School of Medicine and Dentistry discussed data from the phase 3b ORATORIO-HAND trial, which tested ocrelizumab’s effect in patients with more advanced primary progressive multiple sclerosis. [WATCH TIME: 5 minutes]

The pediatrician in chief at Johns Hopkins Children’s Center provided clinical insights on new findings demonstrating ocrelizumab’s potential in treating pediatric multiple sclerosis. [WATCH TIME: 4 minutes]

Gavin Giovannoni, MBBCh, PhD, FCP, FRCP, FRCPath, lead investigator of the phase 3 ORATORIO-HAND trial, provided comment on the results presented at ECTRIMS 2025, highlighting ocrelizumab’s therapeutic benefit in more advanced forms of multiple sclerosis.

Trofinetide shows significant real-world improvements in Rett syndrome symptoms for pediatric and adult patients, enhancing communication and quality of life.

The pediatrician-in-chief at Johns Hopkins Children’s Center shared her reaction to receiving this year’s Bernard Sachs Award in recognition of her contributions to child neurology. [WATCH TIME: 5 minutes]

Experts shared their clinical perspectives on trending topics in the treatment and management of multiple sclerosis at the 41st Congress of the European Committee for Treatment and Research in Multiple Sclerosis.

A case series reveals safe use of vamorolone and prednisolone during microdystrophin gene therapy for Duchenne muscular dystrophy in two boys.

A recent study reveals the significant economic burden of Angelman syndrome, highlighting rising healthcare costs and ongoing therapeutic developments.

At CNS 2025, the executive director at the Child Neurology Foundation presented on the importance of collaboration among clinicians and advocacy groups to connect families with care support services. [WATCH TIME: 4 minutes]

A post hoc analysis of the phase 3 EPIDYS trial presented at CNS 2025 revealed that givinostat delayed disease progression in 2 distinct age groups of patients with Duchenne muscular dystrophy.

A recent analysis highlights the link between vastus lateralis fat fraction and treatment outcomes in Duchenne muscular dystrophy patients using givinostat.

Joseph Vithayathil, MD, discusses being awarded the Elterman Research Grant at the 2025 CNS Annual Meeting in Charlotte, North Carolina.

The associate professor and clinician-scientist at the University of Toronto spoke to the current understanding of protein waste clearance in neurodegeneration. [WATCH TIME: 4 minutes]

The BeeLine trial is a placebo-controlled study designed to evaluate radiprodil’s impact on both seizure frequency and non-seizure symptoms associated with GRIN-NDD.

New data reveals bemdaneprocel, an innovative cell therapy for Parkinson disease, shows promising safety and efficacy over 36 months, paving the way for future trials.

A new phase 2 trial explores probiotics' potential to alleviate depression and mood disorders in patients with Parkinson disease, linking gut health to mental well-being.

The neurologist at University of British Columbia discussed how abnormal brain plasticity influences Parkinson progression and highlighted the importance of integrating basic science with clinical care.

Results from the extension phase of the IB1001-203 study, presented as a late-breaker at MDS 2025, suggest N-acetyl-L-leucine prevented worsening of disease progression in patients with ataxia-telangiectasia.

New heatmap analysis reveals continuous apomorphine infusion significantly improves ON time and reduces OFF episodes in patients with Parkinson disease.

In the MOXIe open-label extension study presented at MDS 2025, omaveloxolone treatment was associated with no significant disease progression in bulbar function or upper limb coordination.

The chief of the Movement Disorders Division at Yale School of Medicine discusses the nuance around patient choice in Huntington disease testing and providing treatment regardless of confirmation. [WATCH TIME: 3 minutes]

The chief medical officer of Gain Therapeutics discusses the challenges that are unaddressed with currently available treatments for Parkinson, and how investigational treatments like GT-02287 might address those needs. [WATCH TIME: 3 minutes]

The neurologist at University of British Columbia discussed how breakdowns in brain organization and compensatory mechanisms may influence progression in Parkinson disease. [WATCH TIME: 5 minutes]