Marco Meglio

The new drug application (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.

Patients aged 2-21 receiving apitegromab showed clinically meaningful motor function improvements, with a favorable safety profile consistent with long-term data, as Scholar Rock prepares for US and EU regulatory submissions in Q1 2025.

The FIREFISH study showed that most children with type I spinal muscular atrophy (SMA) maintained or improved motor functions and feeding abilities over 5 years when treated with risdiplam.

Givinostat, a histone deacetylase inhibitor from Italfarmaco, was supported by data from the phase 3 EPIDYS study, a randomized, double-blind, placebo-controlled, multicenter trial investigating the agent vs placebo.

Results showed that evobrutinib did not produce a more superior reduction in annualized relapse rates than teriflunomide, a previously approved disease-modifying agent.

Over a 52-week treatment period, the gene therapy demonstrated robust evidence for a clinically meaningful benefit, including across several prespecified functional secondary end points vs placebo.

Patients with multiple sclerosis (MS) showed cognitive improvements with frontal theta-transcranial alternating current stimulation (tACS), suggesting its potential as a tolerable and beneficial intervention.

The reported neurofilament light elevation preceding confirmed disability worsening events highlighted the value of NfL as an early biomarker of disability worsening and points to the existence of different windows of dynamic central nervous system pathology.

A retrospective study presented at the 2023 American Association of Neuromuscular and Electrodiagnotsic Medicine meeting revealed that social determinants of health (SDOH) are linked to poorer treatment outcomes in patients who have myasthenia gravis, emphasizing the need for identifying and supporting at-risk individuals.

After 1 year of treatment with risdiplam, 81% of the 26-infant cohort was able to sit independently for 30 seconds, a milestone not typically reached in spinal muscular atrophy (SMA).

With the approval, zilucoplan becomes the first once-daily subcutaneous complement C5 inhibitor available for adults with generalized myasthenia gravis.

Following the switch from natalizumab, patients with multiple sclerosis on anti-CD20 therapies like rituximab demonstrated significant reductions in annualized relapse rate.

Treatment with diazepam nasal spray resulted in a statistically significant change in time between seizure clusters, potentially reflecting a beneficial effect of intermittent rescue therapy.

One meta-analysis featuring 8 studies showed that more than half of patients reported a 100% reduction in their generalized tonic-clonic seizures or tonic-clonic seizures.

Patients treated for more than 28 days had a higher chance of status epilepticus cessation, but also a high risk of moderate to severe disability at discharge, according to one study.

With the decision, ClarityPro becomes the first and only device to offer new technology add-on payment reimbursement for the diagnosis of electrographic status epilepticus.

Stroke of an undetermined cause was associated with acute symptomatic seizures, an effect that was marginally mediated by cerebral microbleeds.

In a 6-month trial, more than 85% of adults on CBD showed significant reduction in the number of monthly seizures, suggesting this treatment may be an effective adjuvant option.

Martina Bebin, MD, MPA, professor of neurology, University of Alabama at Birmingham Epilepsy Center, discussed Infantile Spasms Awareness Week, the strides made within the field, and the emphasis on early and accurate diagnosis.