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1 week ago | 
praderwillinews.com | Lindsey Shapiro
Parents and siblings of people with Prader-Willi syndrome (PWS) commonly experience stress and other mental health challenges, but greater family cohesion, or bonding, helped buffer against these adverse outcomes, according to a study that surveyed people from around the world. In questionnaires completed by parents, parental depression, anxiety, and stress were common, while symptoms of post-traumatic stress disorder (PTSD) were prevalent in siblings.
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1 week ago | 
myastheniagravisnews.com | Lindsey Shapiro
The recently approved prefilled syringe formulation of Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) that offers the possibility of self-administration is now available in the U.S. for eligible adults with generalized myasthenia gravis (gMG) who have antibodies against acetylcholine receptors (AChRs).
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1 week ago | 
pulmonaryhypertensionnews.com | Lindsey Shapiro
The first patient has been enrolled in a pivotal study testing the accuracy of Sensydia’s noninvasive Cardiac Performance System (CPS) for measuring cardiovascular function, including pulmonary artery pressure, a key metric used to diagnose and monitor pulmonary hypertension (PH). The multi-center, observational trial (NCT06870591) will compare CPS to right heart catheterization (RHC), an invasive gold-standard test used to measure pulmonary artery pressure.
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1 week ago | 
epidermolysisbullosanews.com | Lindsey Shapiro
The European Commission has approved the topical gene therapy Vyjuvek (beremagene geperpavec) to treat wounds, starting at birth in people with dystrophic epidermolysis bullosa (DEB) who have disease-causing mutations in the COL7A1 gene. The decision, which applies to all European Union countries, and includes Iceland, Norway, and Liechtenstein, makes Vyjuvek the first corrective medicine that’s designed to address the disease’s root genetic cause to be approved in Europe for DEB.
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2 weeks ago | 
myastheniagravisnews.com | Lindsey Shapiro
The U.S. Food and Drug Administration (FDA) has approved nipocalimab as a treatment for adults and adolescents, ages 12 and older, with generalized myasthenia gravis (gMG) who have antibodies against the acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) proteins. The newly approved therapy will be sold by developer Johnson & Johnson (J&J) under the brand name Imaavy. This is the first regulatory approval for Imaavy in gMG.
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2 weeks ago | 
liverdiseasenews.com | Lindsey Shapiro
Daily treatment with Ipsen’s elafibranor safely and effectively eases itching, stabilizes liver scarring, and reduces liver damage biomarkers in adults with primary sclerosing cholangitis (PSC), according to new data from a Phase 2 clinical trial. Elafibranor is already approved in the U.S. and elsewhere under the brand name Iqirvo for treating certain adults with primary biliary cholangitis (PBC), another rare liver disease characterized by chronic cholangitis.
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2 weeks ago | 
alsnewstoday.com | Lindsey Shapiro
The Muscular Dystrophy Association (MDA) will celebrate the 25th anniversary on June 25 of its Wings Over Wall Street Gala, an annual fundraiser to support research for amyotrophic lateral sclerosis (ALS). The event will take place at the Edison Ballroom in New York City, starting at 6 p.m. ET, and includes a cocktail reception, dinner, and an awards program to recognize leaders in ALS research and advocacy.
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2 weeks ago | 
parkinsonsnewstoday.com | Lindsey Shapiro
Researchers at Emory University and the Georgia Institute of Technology are developing an artificial intelligence (AI)-driven approach to track and measure freezing of gait (FOG), a common motor symptom of Parkinson’s disease. The technology will be used in a clinical trial that’s testing the safety and preliminary efficacy of sound and light stimulation, known as gamma sensory flicker, as a therapeutic approach for Parkinson’s.
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2 weeks ago | 
liverdiseasenews.com | Lindsey Shapiro
Rare mutations in the MET gene can cause metabolic dysfunction-associated steatotic liver disease (MASLD), a form of fatty liver disease, a study found. After identifying a specific disease-causing mutation in a woman and her father who had no common risk factors of the disease, the scientists looked at a larger genetic dataset and found other, similar cases where MET mutations appeared to be likely disease drivers.
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2 weeks ago | 
hemophilianewstoday.com | Lindsey Shapiro
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Hympavzi (marstacimab) for the prevention of bleeding episodes in people with both hemophilia A and hemophilia B, ages 12 and older. The therapy, already approved in the U.S. and the European Union, is designed to prevent or reduce the frequency of bleeds and can be administered by caregivers or patients following adequate training.
