Drug for Rare Form of ALS Earns FDA Nod
The FDA granted accelerated approval to tofersen (Qalsody) for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, the agency announced on Tuesday. The drug was cleared under the accelerated approval pathway, a program that uses a surrogate endpoint to approve drugs for serious conditions with an unmet medical need. For tofersen, the […]
Investigational ALS Drug May Have Clinical Benefit, FDA Staff Says
Tofersen, an investigational antisense drug developed to treat amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene (SOD1-ALS), may have clinical benefit, FDA reviewers indicated in briefing documents ahead of an advisory committee meeting. On Wednesday, the Peripheral and Central Nervous System Drugs Advisory Committee will vote on whether evidence supporting a […]
Pragmatic Trials May Help Increase Minority Participation in MS Research
Pragmatic trials may offer one way to increase minority populations in multiple sclerosis (MS) studies, investigators for the DELIVER-MS trial suggested. Race and ethnicity did not appear to influence decisions to participate in either the randomized or observational cohorts of the pragmatic DELIVER-MS study, or in treatment choices, reported Sarah Planchon, PhD, of the Cleveland […]
No Excess Pregnancy, Infant Risks With Ocrelizumab for MS
Women with multiple sclerosis (MS) who were treated with ocrelizumab (Ocrevus) had no increased risk of adverse pregnancy and infant outcomes, prospective data showed. Cumulative data did not suggest an increased risk of preterm birth, major congenital anomalies, or other adverse outcomes with ocrelizumab in utero exposure and were consistent with epidemiological data, reported Celia […]
Muscle biopsy test for biomarker could lead to earlier diagnosis of ALS
Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system. It affects nerve cells in the brain and spinal cord called motor neurons. Motor neurons control muscle movement and ALS causes them to deteriorate and eventually die. The motor neurons lose the ability to send messages to the muscles in the body, affecting […]
Deep Learning Identifies PPMS Patients Who May Respond to Treatment
Deep learning identified subgroups of patients with primary progressive multiple sclerosis (PPMS) who may respond to disease-modifying therapy, researchers said. A trained model separated responders and non-responders across a range of predicted effect sizes, reported Jean-Pierre Falet, MD, of McGill University in Montreal, Canada, at the ACTRIMS Forum 2022, the annual meeting of the Americas […]
Paramagnetic Rim Lesions Highly Specific in MS Diagnosis
The presence of paramagnetic rim lesions (PRLs) on MRI was highly specific for multiple sclerosis (MS) in people suspected to have the disease, a prospective multicenter study showed. Based on 2017 McDonald criteria for MS diagnosis, the presence of one or more PRLs showed 88% specificity (95% CI 79-98%), 80% sensitivity (95% CI 68-91%), and […]
COVID Vax Response Still Impaired in Some MS Patients After Third Shot
Immune response was attenuated after a third COVID-19 vaccine dose for multiple sclerosis (MS) patients taking certain disease-modifying treatments, interim data from a longitudinal study showed. Two weeks after a third shot, many MS patients on anti-CD20 drugs and sphingosine-1-phosphate (S1P) receptor modulators still had significantly reduced total spike and receptor-binding domain (RBD) antibody responses, […]
Worse COVID-19 Outcomes for Disabled MS Patients
Multiple sclerosis (MS) patients who were disabled were more likely to be hospitalized, end up in the intensive care unit (ICU) or on a ventilator, or die if they developed COVID-19, North American registry data showed. Of non-ambulatory MS patients with COVID-19, 14.0% died, 8.3% were in the ICU or on a ventilator, and 24.6% […]
Even When Undergoing Treatment, People With MS Gain From COVID Vaccines
Latest Neurology News THURSDAY, Sept. 16, 2021 (HealthDay News) Multiple sclerosis (MS) patients undergoing a treatment that depletes a type of immune cell that fuels MS attacks still have a strong response to mRNA COVID-19 vaccines, a new study finds. “The message from this study is clear — it is worthwhile for patients […]