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3 weeks ago | 
epidermolysisbullosanews.com | Lindsey Shapiro
Traws Pharma said it will advance development of skin cancer therapy rigosertib, after data showed the therapy led to a partial or complete treatment response for four recessive dystrophic epidermolysis bullosa (RDEB) patients with advanced forms of the skin cancer squamous cell carcinoma (SCC).
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3 weeks ago | 
multiplesclerosisnewstoday.com | Lindsey Shapiro
Artificial intelligence (AI)-powered algorithms to analyze data collected on a smartphone app could predict whether a person with multiple sclerosis (MS) will experience certain high-severity symptoms in the next three months, a study shows. The scientists believe this will help empower patients to better understand their disease and work more easily with their healthcare team to make disease management decisions.
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3 weeks ago | 
fabrydiseasenews.com | Lindsey Shapiro
Serious complications like cardiovascular disease and other heart issues, kidney problems, and stroke are common among adults with Fabry disease and may be associated with factors such as sex, age at diagnosis, genetic profiles, and cardiometabolic risk factors like obesity, according to the findings of a 20-year U.K. study. Slightly less than half of all reported deaths among these Fabry patients were related to cardiovascular problems like heart failure or heart attack, the data showed.
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3 weeks ago | 
ancavasculitisnews.com | Lindsey Shapiro
Immune neutrophils and the weblike structures they release to trap and kill microbes — called neutrophil extracellular traps, or NETs — are key players in driving kidney damage in ANCA-associated vasculitis (AAV) and other diseases, a review study found. This points to neutrophils and NETs as potential targets for more effective and safer treatments for these conditions, according to the researchers.
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3 weeks ago | 
huntingtonsdiseasenews.com | Lindsey Shapiro
uniQure plans to submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) early next year seeking approval of its gene therapy candidate AMT-130 for Huntington’s disease. Top-line, three-year data from the ongoing clinical trials that will support that application are due in the next few months, and uniQure plans to hold a pre-submission meeting with the agency by year’s end.
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1 month ago | 
liverdiseasenews.com | Lindsey Shapiro
The global prevalence of primary sclerosing cholangitis (PSC), or the total number of existing cases, is expected to increase substantially over the next several years, according to recent projections. Pooled study data suggest the highest prevalence of PSC was seen in the Australasia region and the lowest in high-income areas of the Asia-Pacific. Accordingly, the most dramatic increases are expected in Australasia in the coming years.
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1 month ago | 
multiplesclerosisnewstoday.com | Lindsey Shapiro
A particular finding on an MRI scan, called the central vein sign (CVS), is sufficient to support a multiple sclerosis (MS) diagnosis without the need for an invasive lumbar puncture, a new U.K. study suggests. The presence of six lesions with a CVS, where a vein is found running through the center of a lesion, has a similar diagnostic accuracy as a spinal tap that tests for oligoclonal bands (OCBs) — a type of MS antibody — in the spinal fluid.
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1 month ago | 
fabrydiseasenews.com | Lindsey Shapiro
A type of bone tissue death called avascular necrosis is a rare and painful complication of Fabry disease that clinicians should be aware of, according to a recent case report. The report described a 41-year-old man with Fabry who experienced severe pain and mobility problems from avascular necrosis, and ended up needing his hips replaced. “We expand awareness of early [avascular necrosis] as a potential new complication of Fabry disease in a young patient,” researchers wrote.
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1 month ago | 
liverdiseasenews.com | Lindsey Shapiro
The investigational treatment imdusiran used as part of combination therapy regimens has led to a functional cure for eight people with chronic hepatitis B in clinical trials, according to an update from its developer Arbutus Biopharma. A functional cure means the hepatitis B virus (HBV), which causes the disease, was suppressed to where antiviral treatments could be discontinued.
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1 month ago | 
musculardystrophynews.com | Lindsey Shapiro
The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne muscular dystrophy (DMD) who were treated when they were 8 or 9 years old, according to new trial analyses.
