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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 | 
parkinsonsnewstoday.com | Margarida Maia |Patricia Inacio |Doc Irish |Andrea Lobo
The Van Andel Institute (VAI) — founded nearly three decades ago to advance biomedical research and science education — is awarding its 2025 Jay Van Andel Award to a University of Pittsburgh professor for his work on environmental factors in Parkinson’s disease. This year’s award will be presented to J. Timothy Greenamyre, MD, PhD, a neurologist and researcher who also serves as the director of the Pittsburgh Institute of Neurodegenerative Diseases.
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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 | 
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 | 
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 | 
smanewstoday.com | Andrea Lobo |Marisa Wexler
Treatment with Spinraza (nusinersen) and physical therapy that focused on posture improved motor skills in a young boy with spinal muscular atrophy (SMA) type 0 and prevented the progression of scoliosis. That’s according to a case report in Japan that demonstrated the boy was able to sit without support for short periods at age 2 and could stand with support when he was 3. The treatment goals at his most recent evaluation were to help him stand alone and walk with support.
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1 week ago | 
parkinsonsnewstoday.com | Margarida Maia |Andrea Lobo
ION859, an injectable treatment being developed by Ionis Pharmaceuticals, was generally safe and well tolerated, and it reduced the levels of LRRK2 — a protein often overactive in Parkinson’s disease — even in patients without mutations in the gene that encodes it.
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1 week ago | 
sicklecellanemianews.com | Steve Bryson |Oluwatosin Adesoye |Esteban Cerezo |Andrea Lobo
Women with sickle cell disease (SCD) have markedly elevated levels of C-reactive protein (CRP), a marker of body-wide inflammation, during the first half of their menstrual cycles, and these variations may contribute to recurrent patterns of vaso-occlusive events (VOEs), complications resulting from blood vessel obstruction that include painful vaso-occlusive crises. a study found.
