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5 days ago | 
pnhnews.com | Steve Bryson
Novelmed Therapeutics’ ruxoprubart prevented red blood cell destruction in treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH), allowing these untreated patients to avoid blood transfusions altogether. That’s according to 12-week interim data from an ongoing Phase 2 trial (NCT05646524), which showed the treatment, administered as a standalone therapy, was safe, well-tolerated, and met all primary efficacy outcome measures.
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1 week ago | 
pulmonaryfibrosisnews.com | Steve Bryson
Nerandomilast, Boehringer Ingelheim’s investigational oral therapy, significantly slowed lung function decline in people with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) after a year of treatment, according to final data from two Phase 3 clinical trials. FIBRONEER-IPF (NCT05321069) enrolled 1,177 IPF patients, while FIBRONEER-ILD (NCT05321082) enrolled 1,176 adults with progressive forms of pulmonary fibrosis other than IPF.
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1 week ago | 
liverdiseasenews.com | Steve Bryson
The cumulative healthcare costs for infants with biliary atresia can increase substantially if they undergo their initial treatment, the Kasai surgery, after 2 months of age, according to an analysis of U.S. private-payer insurance claims. Higher healthcare costs in the first year of life was also found for infants who had been admitted to neonatal intensive care and who reside in rural areas compared with those who live in urban centers, data show.
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1 week ago | 
myastheniagravisnews.com | Steve Bryson
Using tears to test for self-reactive antibodies to help diagnose ocular myasthenia gravis (OMG) is more accurate than using blood samples, according to a study that found nearly all patients who tested negative for these antibodies in the blood had a positive result in tears. “Tear-based testing offers a promising alternative for diagnosing [blood]-negative OMG cases,” the researchers wrote.
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1 week ago | 
multiplesclerosisnewstoday.com | Steve Bryson
Clemastine fumarate, an antihistamine, boosted disease progression by more than five times in adults with progressive multiple sclerosis (MS), according to data from a Phase 1/2 trial. “Likely nobody in the MS field, us included, knew about this potential [clemastine] toxicity until we collected the data presented here,” the researchers wrote.
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1 week ago | 
rettsyndromenews.com | Steve Bryson
Girls with Rett syndrome have significantly smaller brain volumes across all its regions, regardless of their age, a MRI study shows. Reductions in the size of certain brain regions were tied to the clinical severity of Rett patients, particularly in gross motor function, such as sitting and walking. Still, scientists found no obvious relationships between brain volume changes and epilepsy.
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1 week ago | 
liverdiseasenews.com | Steve Bryson
ACER3, an enzyme involved in the metabolism of fat-like signaling molecules known as ceramides, plays a detrimental role in cholestasis-related liver damage — and, as such, may represent a new therapeutic target, according to a laboratory study using mice and human cells. Elevated ACER3 levels were found in the liver tissue of people with cholestasis, with higher levels of the enzyme associated with more severe liver damage, per the study.
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1 week ago | 
huntingtonsdiseasenews.com | Steve Bryson
Researchers have developed a modified and harmless adeno-associated virus (AAV), called AAV-DB-3, that can efficiently deliver low doses of gene therapies to deep regions of the brain impacted by neurodegenerative diseases such as Huntington’s disease. AAV-DB-3 was more than 200 times more potent at delivering its genetic cargo to brains of nonhuman primates (NHPs) than other AAV-based gene therapies now being assessed in Huntington’s.
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2 weeks ago | 
cysticfibrosisnewstoday.com | Steve Bryson
Scientists have discovered a potential therapy for cystic fibrosis (CF) patients who are infected with Mycobacterium abscessus, a bacterium that can cause serious lung infections. They found that a signaling molecule called PI5P can boost the antimicrobial activity of immune cells isolated from patients against the bacterium. This happens even for cells from patients not eligible for treatment with Kaftrio, a CFTR modulator therapy that’s marketed in the US. as Trikafta.
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2 weeks ago | 
myastheniagravisnews.com | Steve Bryson
Enrollment is open in an investigator-initiated clinical trial to evaluate cell therapy NKX019 in people with myasthenia gravis (MG). Nkarta, the therapy’s developer, made the announcement in a company press release. Researchers at the University of California, Irvine, and the University of Kansas Medical Center are leading the open-label Phase 1 trial, which was given the go-ahead by the U.S. Food and Drug Administration late last year.
