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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 | 
angioedemanews.com | Marisa Wexler |Danita Jones |Andrea Lobo |Margarida Maia
A simple diagnostic score may help distinguish between two types of angioedema — mast cell-mediated angioedema and drug-induced nonallergic angioedema — in people taking angiotensin-converting enzyme (ACE) inhibitors, researchers said. They described the scoring system in the study, “Angiotensin-converting enzyme inhibitor-induced angioedema: Proposal for a diagnostic score,” published in the World Allergy Organization Journal.
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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 | 
multiplesclerosisnewstoday.com | Margarida Maia |Benjamin Hofmeister |Marisa Wexler
Biostate AI is partnering with the nonprofit Accelerated Cure Project (ACP) to develop a series of artificial intelligence (AI) models that can predict multiple sclerosis (MS) progression and how patients may respond to treatment. As part of the partnership, Biostate AI will use its high-throughput technology to profile RNA reads from ACP’s large collection of MS patient samples. RNA reads can be used as a measure of how active different genes are.
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1 week ago | 
musculardystrophynews.com | Marisa Wexler |Patrick Moeschen |Lindsey Shapiro
The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies already in clinical testing for other types of LGMD.
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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 | 
ancavasculitisnews.com | Margarida Maia |Marisa Wexler |Patricia Inacio
Elevated levels in the blood of ferritin, a protein that binds to iron and stores it for use by the body, are linked to more severe damage in people with active ANCA-associated vasculitis (AAV), and to a poorer outlook for the kidneys, according to a new study from China. An analysis by researchers showed that a “high [blood ferritin] level was a risk factor for [end-stage renal (kidney) disease] and death,” the team wrote.
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1 week ago | 
parkinsonsnewstoday.com | Margarida Maia |Marisa Wexler |Andrea Lobo
The investigational liquid therapy ND0612 — an around-the-clock supply of levodopa/carbidopa infused via a pump under the skin — provides more stable motor control in people with Parkinson’s disease who experience motor fluctuations than standard immediate-release levodopa/carbidopa. That’s according to data from the randomized portion of the Phase 3 BouNDless (NCT04006210) study and its ongoing open-label extension.
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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 | 
ahusnews.com | Marisa Wexler
Plasma exchange therapy followed by treatment with Soliris (eculizumab) was effective at managing atypical hemolytic uremic syndrome (aHUS) in a boy in China. The case was described in the report, " Plasma exchange combined with eculizumab in the management of atypical hemolytic uremic in pediatric patients: A case report ," which was published in Medicine.
