Marco Meglio

Branded as Qalsody, the FDA has granted approval to Biogen and Ionis' tofersen, making it the fourth therapy approved for ALS and the first for SOD1-associated disease.

No significant differences between P2B001 and Prami-ER were seen in total UPDRS responders, UPDRS Part II, and UPDRS Part III scores among those with Parkinson disease.

Adverse events like dyskinesia, ON and OFF phenomenon, and falls, were all higher during the 4-week optimization phase than the 8-week maintenance phase.

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.

After 228 weeks of treatment, annualized relapse rates favored twice-daily 75 mg dosing of evobrutinib vs once-daily.

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

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.

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.

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.

After 90 days, investigators found that tenecteplase was not associated with improvements in functional outcome, nor did it differ from placebo on mortality.

NMOSD-related optic neuritis represented the strongest predictive risk factor of failure to attain visual recovery of at least 0.3 logMAR, with an odds ratio of 10.47.

Greater cumulative exposure to HbA1c concentrations in the range of 6% to less than 8%, targets proposed by most geriatrics’ guidelines for healthier older people, were associated with lower hazard of dementia.

After showing promise in a phase 2 assessment, Taisho Pharmaceutical’s TS-142 will be assessed in a long-term study of 300 patients with the sleeping disorder.

Collectively, the results suggested that multiple sclerosis and migraine may co-occur because they share several genetic variants, rather than migraine causing MS.

The director of the Memory & Cognitive Disorders Clinic at Hoag Neuroscience Institute provided detail on an educational course that examined medication management for Parkinson disease psychosis.

In a study of more than 1500 adolescents, two-thirds of individuals experienced relief from functional disability with Nerivio.

Fenfluramine was originally approved as a therapy for Dravet syndrome in 2020, and later gained expanded indication for Lennox-Gastaut syndrome in 2022.

The FDA is expected to make a decision by May 10, 2023, whether brexpiprazole will become the first approved therapy for Alzheimer disease agitation.

A derived Obstructive Sleep Apnea score of 2-3 and cumulative sleep symptoms were found to be associated with a significantly higher odds of acute stroke.

In addition to high rates of individuals who required rescue therapy after midazolam, a substantial number of children needed respiratory support following.

In patients with high C-reactive protein levels treated with NP001, investigators observed a 64% slower rate of decline in forced vital capacity.

ARIA-inducing monoclonal antibodies accelerated ventricular enlargement to a point where a striking correlation between ventricular volume and ARIA frequency was observed.

The multicenter, multiple-ascending dose trial will feature 64 patients with ALS who will be assessed on safety and pharmacokinetic outcomes.

Evobrutinib, a highly selective BTK inhibitor, has previously demonstrated positive impacts on relapse rates and neurofilament light in patients with relapsing multiple sclerosis.

On average, across all levels of physical fatigue, participants would accept a 0.32 year decrease in time to multiple sclerosis progression in exchange for a 3.57-point change in FSIQ-RMS-S score.

The neurosurgeon at Marcus Neuroscience Institute, Baptist Health, provided perspective on invasive procedures for degenerative conditions and when deep brain stimulation is needed.

In the phase 2 study, patients with Guillian-Barré syndrome will receive imlifidase on day 1, followed with intravenous immunoglobulin on days 3-7, with outcomes of safety and change in disability.

Compared with children without posttraumatic headache, those who experienced migraine symptoms following concussion were nearly 3 times more likely to report reliable increases in somatic symptoms.

At 2-year follow-up, 72% of patients with drug-resistant epilepsy who underwent surgery were seizure-free, compared with 33% of patients on standard medical therapy.

After 6 months of stimulation, more than half (53%) of patients with drug-resistant epilepsy were responders to treatment with at least a 50% reduction in seizure frequency.