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6 days ago | 
multiplesclerosisnewstoday.com | Marisa Wexler
In people with relapsing forms of multiple sclerosis (MS) treated with Ocrevus (ocrelizumab), levels of B-cells can help to predict the risk of progression independent of relapse activity, a new study reports. “Our results unveil a discernible relationship between the degree of B-cell depletion and disease progression in individuals with [relapsing] MS,” researchers wrote.
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1 week ago | 
angioedemanews.com | Marisa Wexler
Biocryst Pharmaceuticals has submitted an application asking the U.S. Food and Drug Administration (FDA) to extend the approval of Orladeyo (berotralstat) to children with hereditary angioedema (HAE) as young as 2 years. The application specifically seeks the approval of an oral granule formulation of the therapy for children ages 2-11. If approved, Orladeyo would become the first targeted oral preventive therapy to be approved for children with HAE.
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1 week ago | 
parkinsonsnewstoday.com | Marisa Wexler
An experimental compound has been identified that shows the potential to reduce brain inflammation in neurodegenerative conditions such as Parkinson’s disease. MFP-0012328 works by blocking FABP4, a protein that researchers have shown to be key in the inflammatory activity of microglia, the brain’s immune cells. While other FABP4 suppressors have been studied, this one appears to show the most pronounced effects and favorable properties for reaching the brain.
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1 week ago | 
rettsyndromenews.com | Marisa Wexler
Unravel Biosciences is asking authorities in Colombia to allow the launch — which could be as early as this summer, if cleared — of proof-of-concept clinical trials in the country to test RVL001, its treatment candidate for Rett syndrome. The company has submitted an application seeking regulatory clearance for a trial of RVL001, involving Rett patients, at the Universidad de Antioquia’s Center for Technological Development in Medellin.
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1 week ago | 
ahusnews.com | Marisa Wexler
Soliris (eculizumab) helped control atypical hemolytic uremic syndrome (aHUS) in a patient whose symptoms included seizures and signs of neurological damage, according to a case report.
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1 week ago | 
multiplesclerosisnewstoday.com | Marisa Wexler
Foralumab was well tolerated in people with nonactive secondary progressive multiple sclerosis (SPMS), according to data from a small, open-label study conducted under an expanded access program. The therapy, being developed by Tiziana Life Sciences, also eased fatigue and lessened disability in some participants. The promising findings formed the basis for a Phase 2a study (NCT06292923) that’s currently testing foralumab against a placebo in adults with nonactive SPMS.
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1 week ago | 
liverdiseasenews.com | Marisa Wexler
With ESP-1336 as their lead candidate, Esperion Therapeutics is developing new treatments for primary sclerosing cholangitis (PSC) by targeting an enzyme called ACLY. “We are leveraging our deep expertise in ACLY biology … to address the urgent needs of patients living with PSC,” which is “a rare and progressive liver disease with no approved therapies proven to slow or halt its progression,” Sheldon Koenig, CEO of Esperion, said in a company press release.
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1 week ago | 
porphyrianews.com | Marisa Wexler
The seven-day Porphyria Awareness Week campaign kicks off May 12 to spread the word about porphyria and culminates with Global Porphyria Day on May 18, a Sunday. The theme of this year’s observance is “Think Porphyria,” according to the American Porphyria Foundation, which is joining forces with other organizations in the Global Porphyria Advocacy Coalition to raise awareness about the disease.
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1 week ago | 
dravetsyndromenews.com | Marisa Wexler
Praxis Precision Medicines is gearing up to launch a clinical trial of its experimental anti-seizure therapy relutrigine in people with Dravet syndrome and other disorders that cause seizures in childhood, collectively known as developmental and epileptic encephalopathies (DEEs).
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1 week ago | 
myastheniagravisnews.com | Marisa Wexler
Enrollment is now complete for a Phase 2 clinical trial testing Dianthus Therapeutics‘ treatment candidate DNTH103 in people with generalized myasthenia gravis (gMG). According to the developer, the initial results from the global study, dubbed MAGIC (NCT06282159), are expected by early fall. MAGIC was launched early last year and is being conducted at 56 locations in North and South America, Europe, and Asia.
