VEXAS Syndrome Often Misdiagnosed as Pyoderma Gangrenosum
July 3rd 2024In a recently published systematic review, researchers determined that VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome typically presents in men, with atypical morphology and with systemic symptoms.
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Final Readout of FIREFISH Study Showcases Risdiplam’s Pronounced Effects in Spinal Muscular Atrophy
July 3rd 2024The FIREFISH study showed that most children with type I spinal muscular atrophy (SMA) maintained or improved motor functions and feeding abilities over 5 years when treated with risdiplam.
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A Breathtaking Crisis, Part 2: Climate Change Worsens Asthma for Underserved Communities
July 3rd 2024Underserved communities, already facing higher asthma prevalence due to social determinants of health, are disproportionately affected by the impact climate change is having on asthma exacerbations. Potential solutions include education for patients and health care providers, climate litigation to establish environmental rights, and policy changes to curb climate change.
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Study Finds Ravulizumab Effective in Treating Acetylcholine Receptor–Positive Generalized MG
July 2nd 2024More than 60% of complement inhibitor–naive patients with acetylcholine receptor–positive generalized myasthenia gravis (MG) in diverse clinical populations had a clinically meaningful reduction in MG activities of daily living scores.
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Insurers continue to raise consumer costs despite covering fewer drugs and having more drug coverage restrictions; the Biden Administration is investing hundreds of millions of dollars to train geriatricians; the Federal Trade Commission has opened an investigation into Teva Pharmaceuticals after the company refused to take down patents for its asthma and chronic obstructive pulmonary disease inhalers.
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Advancements and Challenges in Treating Relapsed/Refractory Multiple Myeloma
July 2nd 2024In part 2 of our interview with Suzanne Lentzsch, MD, PhD, Columbia University's College of Physicians and Surgeon, she touches on potential new therapies, important clinical considerations, and treatment challenge for relapsed/refractory multiple myeloma.
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AAV Therapy: A Successful 1-Patient SPG50 Gene Therapy Trial
July 1st 2024An adeno-associated virus (AAV) gene therapy for hereditary spastic paraplegia type 50 (SPG50)—funded by a young patient’s family—gives hope not only to a 4-year-old, but to researchers working on similar projects for other rare diseases.
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