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1 week ago | 
alsnewstoday.com | Steve Bryson |Patricia Inacio |Esteban Cerezo |Lindsey Shapiro
An experimental candidate by Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS) linked to mutations in the SOD1 gene has shown promising signs of slowed functional decline and stable or improved breathing in ALS patients of a small early trial. The therapy, RAG-17, also significantly reduced blood levels of neurofilament light chain (NfL), a biomarker of nerve damage, and demonstrated target engagement, as indicated by a drop in SOD1 protein levels.
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1 week ago | 
sicklecellanemianews.com | Steve Bryson |Oluwatosin Adesoye |Esteban Cerezo |Andrea Lobo
Women with sickle cell disease (SCD) have markedly elevated levels of C-reactive protein (CRP), a marker of body-wide inflammation, during the first half of their menstrual cycles, and these variations may contribute to recurrent patterns of vaso-occlusive events (VOEs), complications resulting from blood vessel obstruction that include painful vaso-occlusive crises. a study found.
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3 weeks ago | 
pulmonaryhypertensionnews.com | Andrea Lobo |Marisa Wexler |Lindsey Shapiro |Esteban Cerezo
Pulmonary hypertension (PH) is associated with an increased risk for sleep disorders, including obstructive sleep apnea, and structural brain changes at the genetic level, according to a recent study. These findings may help “develop new guidelines for the clinical management, personalized diagnosis, and treatment of PH, sleep disorder diseases, and their associated neurological comorbidities [coexisting disorders],” researchers wrote.
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4 weeks ago | 
praderwillinews.com | Margarida Maia |Vanda Pinto |Steve Bryson |Esteban Cerezo
The U.S. Food and Drug Administration (FDA) has approved Soleno Therapeutics’ extended-release formulation of diazoxide choline (DCCR) for easing hyperphagia — marked by insatiable hunger — in Prader-Willi syndrome (PWS) patients ages 4 and older. The newly authorized oral therapy will be marketed under the brand name Vykat XR.
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1 month ago | 
hemophilianewstoday.com | Andrea Lobo |Esteban Cerezo |Margarida Maia |Jennifer Lynne
About 30% of people hospitalized due to acquired hemophilia have a bleeding event, while 17% report a thrombotic, or blood clot-related complication, such as stroke and deep vein thrombosis, a nationwide U.S. study finds. Patients ages 65 years and older, and those with thrombotic events were more likely to die during a hospitalization. Also, about a third were readmitted within 30 days of being discharged, mainly due to bleeding events and infections.
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1 month ago | 
myastheniagravisnews.com | Sarah Bendiff |Andrea Lobo |Esteban Cerezo |Shawna Barnes
Seventeen years of living with myasthenia gravis (MG) has made me a pro at navigating daily life. I’ve learned how to handle both routine tasks and high-pressure days because being sick doesn’t mean I get to sit out on life. Over the years, I’ve worked on big projects, organized events, and fought hard for my education. Through all of it, I’ve picked up some solid survival strategies to help me keep going, even when MG tries to slow me down. I admit that I’m easily excited.
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1 month ago | 
multiplesclerosisnewstoday.com | Marisa Wexler |Lindsey Shapiro |Esteban Cerezo |Leigh Nelson
Artificial intelligence (AI) platforms did better than most neurologists at answering a 20-question assessment about multiple sclerosis (MS) in a recent study, suggesting that AI may be a helpful tool for MS care. Neurologists with an MS specialty scored as well, on average, as the AI platforms, while neurology residents with less than two years of training had the lowest scores.
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1 month ago | 
fapnewstoday.com | Margarida Maia |Esteban Cerezo |Patricia Inacio
Wainzua (eplontersen) is now approved in the European Union (EU) for treating adults with early-stage familial amyloid polyneuropathy (FAP). With the European Commission’s approval, Wainzua can treat adults in the first two stages of FAP — stage 1, when abnormal sensations appear, but the patient is still able to walk unaided, and stage 2, when the patient can walk, but needs help.
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1 month ago | 
alsnewstoday.com | Lindsey Shapiro |Marisa Wexler |Margarida Maia |Esteban Cerezo
Treatment with CNM-Au8 in the HEALEY ALS platform trial prolonged survival for people with amyotrophic lateral sclerosis (ALS) relative to those who received the investigational treatment zilucoplan or a placebo in a separate arm of the study, according to new analyses. The benefits were most pronounced in the subgroup who meet the enrollment criteria for the planned confirmatory Phase 3 RESTORE-ALS trial, set to launch mid-year.
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1 month ago | 
multiplesclerosisnewstoday.com | Marisa Wexler |Shana Gray |Esteban Cerezo |Lindsey Shapiro
In the lymph nodes of the neck of people with multiple sclerosis (MS), certain immune cells show abnormalities in activity and signs of reactions against the Epstein-Barr virus (EBV), a new study reports. The findings may help to explain the connection between MS and EBV, researchers said. The study, “Altered immune landscape of cervical lymph nodes reveals Epstein-Barr virus signature in multiple sclerosis,” was published in Science Immunology.
