Isabella Ciccone, MPH

The FDA has recently granted approval to Soleno Therapeutics' DCCR tablets as the first treatment for hyperphagia in Prader-Willi syndrome.

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.

The senior vice president of research at The ALS Association discussed insights into neural regeneration therapies and their potential to restore function in neurodegenerative diseases.

Topline data from the REFOCUS-ALZ trial showed no significant cognitive or functional benefits with simufilam in patients with mild-to-moderate AD, leading the company to discontinue the program.

The assistant professor in the department of neurology at Mount Sinai discussed distinguishing cognitive impairment in MS from AD emphasizing orientation as a key differentiator. [WATCH TIME: 5 minutes]

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.

The professor of neurology at University of Colorado School of Medicine talked about the challenges of telehealth and device-based data collection in clinical practice. [WATCH TIME: 4 minutes]

Findings from a phase 2 study of nomlabofusp, a novel investigational therapy for Friedreich ataxia, demonstrated an increase in tissue frataxin levels and metabolic improvements.

Despite advancements in NMOSD treatments, the founder and executive director of the Sumaira Foundation discussed how misdiagnosis, delayed diagnosis, and disparities in care remain significant challenges. [WATCH TIME: 4 minutes]

Results from a recent phase 3 study presented at the 2025 MDA Conference reported that oral edaravone remained well tolerated in patients with ALS over 96 weeks, with no new safety concerns.

The professor of neurological sciences at the University of Vermont discussed the work of the MS Differential Diagnosis Consortium, highlighting efforts to refine MS differential diagnosis and improve clinical accuracy. [WATCH TIME: 8 minutes]

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.

The medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital discussed findings from phase 3 trials suggesting that baseline PRLs can predict disability accumulation and may serve as biomarkers for treatment response to tolebrutinib. [WATCH TIME: 6 minutes]

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.

The company recently announced positive topline results from its phase 3 study in patients with MG and initial data from its phase 2b study in patients with CIDP, both assessing batoclimab.

The design of a phase 2 trial to investigate the potential of satralizumab in boys with Duchenne muscular dystrophy was recently presented at the 2025 MDA Clinical & Scientific Conference.

The phase 3b STRENGTH study reported that a single dose of OAV101IT was well tolerated in treatment-experienced patients with spinal muscular atrophy, with motor function stabilizing over 52 weeks.

Real-world findings from the RESTORE registry presented at the 2025 MDA conference confirmed the established safety profile of onasemnogene abeparvovec over a 5-year period.

Long-term follow-up data from an early-phase study of an AAV8-based gene therapy for DMD suggest the treatment was well-tolerated and provided sustained biochemical and functional benefits.

Alpha Cognition has officially launched its new treatment for mild to moderate Alzheimer disease, with a focus on expanding patient access in the long-term care market.

Findings from the phase 2 FIGHT DMD trial suggest an investigational oral thromboxane prostanoid receptor antagonist may preserve heart function in patients with DMD-associated cardiomyopathy.

A recent study shows that eteplirsen, a drug promoting dystrophin production, significantly slowed the decline in heart function, specifically left ventricular ejection fraction, in patients with Duchenne muscular dystrophy.

Experts in sleep medicine discuss the detrimental effects of daylight saving time on sleep, health, and public safety, advocating for the adoption of permanent standard time. [WATCH TIME: 5 minutes]

Experts discussed the negative health and societal effects of daylight saving time, advocating for a permanent switch to standard time to improve sleep, well-being, and productivity. [WATCH TIME: 6 minutes]

Experts discussed the biological misalignment caused by daylight saving time, emphasizing its negative effects on sleep, particularly in adolescents, and advocating for a shift to standard time to better align with natural circadian rhythms. [WATCH TIME: 6 minutes]

Experts discussed the effects of daylight saving time on sleep patterns, cognitive function, mood, and overall health, highlighting challenges and adaptation strategies. [WATCH TIME: 6 minutes]

Experts discussed how daylight saving time affects sleep patterns, particularly in individuals with insomnia and other sleep disorders, highlighting the challenges of adaptation and the risks of chronic sleep loss. [WATCH TIME: 5 minutes]

Findings from a recently published large-scale analysis highlighted the high burden of cognitive impairment among veterans with both migraine and TBI.

The interim chief executive officer at the Consortium of Multiple Sclerosis Centers invites healthcare professionals to the Consortium’s annual meeting from May 28–31 in Phoenix, Arizona. [WATCH TIME: 2 minutes]

Participants who received the new batch of capsules during the extension phase experienced a lower incidence of falls compared with those taking the old capsules or placebo.