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1 week ago | 
sicklecellanemianews.com | Lindsey Shapiro |Andrea Lobo |Marisa Wexler |Steve Bryson
The U.S. Patent Office has given AB Science notice that it will allow a patent to be issued covering the use of the experimental therapy masitinib for sickle cell disease (SCD). This medical use patent will be valid until November 2040, giving AB Science exclusive rights to develop masitinib for SCD until that time. A similar patent application was approved in Europe late last year, also covering the treatment candidate through 2040.
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1 week ago | 
liverdiseasenews.com | Lindsey Shapiro |Steve Bryson |Marisa Wexler
The U.S. Food and Drug Administration (FDA) has granted fast track status to PBGENE-HBV, Precision Bioscience’s gene-editing therapy candidate for chronic hepatitis B — now being tested in patients in a Phase 1 clinical trial. Fast track status aims to expedite the clinical development and regulatory review of treatments with the potential to address an unmet medical need for serious or life-threatening conditions.
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1 week ago | 
fabrydiseasenews.com | Margarida Maia |Lindsey Shapiro |Steve Bryson |Susanna VanVickle
A woman with heart failure due to late-onset Fabry disease received a heart transplant in combination with immunosuppressants and Fabrazyme (agalsidase-beta) to resolve her cardiac symptoms, which didn’t recur after the transplant. “Although the risk of disease recurrence in the transplanted organ appears to be relatively low, this observation requires further investigation with extended follow-up and a larger study sample,” wrote researchers in Poland.
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1 week ago | 
rettsyndromenews.com | Andrea Lobo |Steve Bryson |Vanda Pinto |Marisa Wexler
A new study highlights how MeCP2 protein dysfunction, particularly related to changes in alternative splicing — a process by which different proteins can be made from the same gene — implicated in brain function, may contribute to Rett syndrome. Specifically, the researchers found that in both Rett patients and mouse models of Rett, there were changes in genes related to synaptic function, or the transmission of nerve signals between cells.
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1 week ago | 
fapnewstoday.com | Margarida Maia |Steve Bryson |Katherine Poinsatte
Here in New Zealand, clinicians and advocates are pushing our country’s government to update its genetic editing and modification legislation. Historically, New Zealand has maintained some of the strictest gene technology regulations globally. Currently, genetic modification is heavily regulated under the . The legislation was intended, in part, to protect the environment and public health from genetically modified organisms, which the act classifies as new organisms.
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1 week ago | 
smanewstoday.com | Helen Baldwin |Steve Bryson |Halsey Blocher |Kevin Schaefer
Years ago, I taught kindergarten at a self-contained school for students with orthopedic and other disabilities. My charges were animated, and my assistant was delightful. At the end of my fifth year, my students were all mainstreamed to regular elementary schools, leaving me classless until days before the following year commenced. Mary, our school nurse, quietly described my new students, all of whom required skilled nursing in some capacity.
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1 week ago | 
porphyrianews.com | Marisa Wexler |Kalyn N Shelton |Steve Bryson |Marta C. Figueiredo
People with acute intermittent porphyria (AIP) often consume large amounts of simple sugars and fats, which may contribute to metabolic problems, such as being overweight or having high levels of bad cholesterol in their blood, a study reports. “Our study provided valuable insights into the metabolic aspects of AIP, revealing improper dietary habits that may predispose patients to additional pathologies [health problems],” the researchers wrote.
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1 week ago | 
multiplesclerosisnewstoday.com | Steve Bryson |Margarida Maia |Andrea Lobo |Leigh Nelson
A single 600 mg dose of Briumvi (ublituximab-xiiy) — instead of the approved 150 mg initial dose plus a 450 mg dose two weeks later — was well tolerated by adults with relapsing forms of multiple sclerosis (MS). That’s according to new data from the ENHANCE Phase 3b clinical trial (NCT05877963), which is evaluating the safety and efficacy of a modified treatment regimen of Briumvi.
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1 week ago | 
cushingsdiseasenews.com | Steve Bryson |Andrea Lobo |Noura Costany |Margarida Maia
Most people with Cushing’s syndrome see their adrenal function recover after first-line surgery to remove disease-causing tumors, even though it may take several months or years, according to a study that also showed a younger age at diagnosis was linked to faster adrenal recovery. “The recovery rate of adrenal function after successful surgery as first-line treatment in patients with Cushing’s syndrome is high,” the researchers wrote.
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1 week ago | 
alsnewstoday.com | Steve Bryson |Patricia Inacio |Esteban Cerezo |Lindsey Shapiro
An experimental candidate by Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS) linked to mutations in the SOD1 gene has shown promising signs of slowed functional decline and stable or improved breathing in ALS patients of a small early trial. The therapy, RAG-17, also significantly reduced blood levels of neurofilament light chain (NfL), a biomarker of nerve damage, and demonstrated target engagement, as indicated by a drop in SOD1 protein levels.
