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4 days ago | 
alsnewstoday.com | Marisa Wexler
Treatment with the experimental therapy jacifusen was generally well tolerated and appeared to slow or even reverse disease progression in some people with amyotrophic lateral sclerosis (ALS) caused by mutations in the FUS gene. That’s according to data from 12 people who received the therapy as part of an expanded access program. Data from these cases also indicate jacifusen decreased nerve damage.
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4 days ago | 
musculardystrophynews.com | Marisa Wexler
Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy for Duchenne muscular dystrophy (DMD). The company said it will wind down all DMD-related research and development activities, focusing instead on an investigational therapy for myotonic dystrophy type 1 (DM1) that’s in clinical testing.
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5 days ago | 
liverdiseasenews.com | Marisa Wexler
The total number of adolescents and young adults with metabolic dysfunction-associated steatotic liver disease (MASLD), a form of fatty liver disease, has been steadily increasing over the last three decades. If that trend continues, more than 16,000 out of every 100,000 young people are expected to have MASLD in 2050, a study shows.
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5 days ago | 
multiplesclerosisnewstoday.com | Marisa Wexler
Pro-inflammatory signaling molecules that have been implicated in multiple sclerosis (MS) cause problems with the growth and development of myelin-making cells in the brain, according to a study done in cell models. The findings may have implications for treating MS, as promoting the growth of myelin-making cells could be a strategy for therapeutic approaches, the researchers said.
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1 week ago | 
liverdiseasenews.com | Marisa Wexler
People with hepatitis C in the U.S. who are insured through state Medicaid programs that require them to be sober to access treatment are more likely to be hospitalized and tend to rack up higher healthcare-related costs, a new analysis demonstrates.
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1 week ago | 
multiplesclerosisnewstoday.com | Marisa Wexler
People who experience adversity during childhood, such as abuse or neglect, may be at increased risk of multiple sclerosis (MS) and other autoimmune diseases, but available data that supports this association is generally low quality, a study reports. “Drawing concrete treatment recommendations is premature, given the low certainty of the findings,” wrote the researchers wrote, who emphasized a need for more studies to explore this potential association.
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1 week ago | 
alsnewstoday.com | Marisa Wexler
A study found that genetic mutations associated with amyotrophic lateral sclerosis (ALS) lead to problems with the function and transport of mitochondria, cellular structures needed for energy production, early in the disease course, before abnormal protein clumps are formed.
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1 week ago | 
ahusnews.com | Marisa Wexler
A woman in Italy developed atypical hemolytic uremic syndrome (aHUS) as a result of a blood cancer, which resolved after the cancer was brought into remission, a case study shows. The report, " Marginal zone lymphoma with anti-factor H IgM and atypical hemolytic uremic syndrome successfully treated with odronextamab ," was published in Haematologica.
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1 week ago | 
sclerodermanews.com | Marisa Wexler
June is Scleroderma Awareness Month, and the scleroderma community is kicking into gear to spread awareness about the rare, chronic disorder. The National Scleroderma Foundation has selected “Stories of Strength” as this year’s theme, encouraging people with scleroderma to share their stories about how the disease has affected them and how they have adapted to living with scleroderma.
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1 week ago | 
myastheniagravisnews.com | Marisa Wexler
A rare form of myasthenia gravis that manifests in childhood and adolescence called juvenile myasthenia gravis (JMG) affects roughly 30 out of every 1 million children in the U.S., and may be more common than previously thought, a study estimates. The study also found that JMG is more common among girls than boys, and in Black children over white children. “Understanding the incidence and prevalence of JMG is crucial for optimizing its management.
