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1 week 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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2 weeks 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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2 weeks 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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2 weeks 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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2 weeks 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.
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2 weeks ago | 
parkinsonsnewstoday.com | Lindsey Shapiro
Exposure to PM10 — air pollution that can come from wildfire smoke, construction dust, and vehicle exhaust — was associated with an increased risk of developing Parkinson’s disease in a recent Italian study. Part of the relationship was explained by blood levels of lipoprotein a, which is a protein that transports fats like cholesterol through the bloodstream.
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3 weeks ago | 
lupusnewstoday.com | Lindsey Shapiro
People with systemic lupus erythematosus (SLE) who have antibodies that target the mitochondrial transcription factor A (TFAM) protein are more likely to develop certain blood clotting complications, a study found. The antibodies were a risk factor for blood clots, or thrombotic events, even when accounting for other factors that could play a role, such as smoking or the presence of other types of self-reactive antibodies (autoantibodies) linked to SLE.
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3 weeks ago | 
liverdiseasenews.com | Lindsey Shapiro
AHB-137, Ausperbio’s experimental therapy, safely and effectively suppressed the disease-causing hepatitis B virus (HBV) in people with chronic hepatitis B, according to interim data from a Phase 2b clinical trial. The positive findings — which are overall consistent with previously reported data from an ongoing Phase 1/2a trial (NCT06115993) — were presented in a poster session at the annual European Association for the Study of the Liver Congress, held last week in Amsterdam and virtually.
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3 weeks ago | 
alsnewstoday.com | Lindsey Shapiro
Clene Nanomedicine is on track to seek accelerated approval of CNM-Au8 for treating amyotrophic lateral sclerosis (ALS) by the end of the year, backed by neurofilament light chain (NfL) biomarker data from its expanded access program (EAP). Those analyses, which are intended to address a recommendation from the U.S. Food and Drug Administration (FDA), are due by early fall.
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3 weeks ago | 
lupusnewstoday.com | Lindsey Shapiro
October 23, 2023 by Lindsey Shapiro Lupus Therapeutics to support development of NK cell therapy Lupus Therapeutics will partner with Nkarta to advance the development of NKX019, Nkarta’s investigational natural killer (NK) cell therapy, for people with treatment-resistant lupus nephritis, a serious complication of lupus that features kidney damage and dysfunction. The U.S. Food and Drug Administration (FDA) recently cleared the launch…
