Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data
May 16th 2025Delandistrogene moxeparvovec demonstrated a manageable safety profile across clinical trials for Duchenne muscular dystrophy (DMD), with most adverse events emerging within 90 days of infusion.
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Specialty Drug Users to Gain Relief Under Medicare Reforms
May 16th 2025A new analysis looked at estimated out-of-pocket costs for etanercept, ustekinumab, and ibrutinib, specialty drugs that often represent a significant cost burden for Medicare Part D beneficiaries and which were selected for 2026 drug price negotiation under the Inflation Reduction Act.
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Dual Therapy Appears to Benefit Patients With Pulmonary Fibrosis, Pulmonary Hypertension
May 15th 2025The combination of antifibrotic therapy and pulmonary vasodilator therapy improved transplant-free survival but had no significant impact on exercise capacity in pulmonary fibrosis and pulmonary hypertension.
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Rozanolixizumab for gMG Approved in Japan for Self-Administration
May 15th 2025The approval marks the second international approval for rozanolixizumab (Rystiggo; UCB Pharma) for generalized myasthenia gravis (gMG), behind the February EU approval of 2 self-administration approvals for the neonatal Fc receptor monoclonal antibody: an infusion pump and manual push with a syringe.
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Infant RSV Hospitalization Rates Drop in First Season With Widespread Preventive Product Use
May 14th 2025New respiratory syncytial virus (RSV) prevention products significantly reduce infant hospitalization rates, highlighting the importance of timely vaccination and nirsevimab administration.
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Future Research Priorities for Non-Hodgkin Lymphomas: Craig A. Portell, MD
May 14th 2025Future research into non-Hodgkin lymphoma treatments needs to explore long-term outcomes and a better understanding of the biologic rationale for various treatments, explained ECHELON-3 principal investigator Craig A. Portell, MD, of UVA Health.
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3 Maternal Risk Factors Linked to Elevated Offspring Blood Pressure
May 14th 2025Offspring born to mothers with prepregnancy obesity, gestational diabetes, or hypertensive disorders of pregnancy had higher systolic and diastolic blood pressure levels than those born to mothers without these risk factors.
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Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression
May 14th 2025Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients with Duchenne muscular dystrophy (DMD) based on muscle quantitative magnetic resonance measures.
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Pediatric Patients With Myasthenia Gravis Need More Treatment Options: Jonathan Strober, MD
May 14th 2025"The newer medications that are coming out, and a lot of them are approved, are much more focused on the problem," explains Jonathan Strober, MD, pediatric neurologist with UCSF Benioff Children's Hospital.
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