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FDA Approves Aqvesme for Thalassemia Anemia, First U.S. Therapy for Both Patient Groups

Approval reflects late-stage efficacy results with use restricted under a REMS requiring frequent liver tests.

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HHS Adds Duchenne Muscular Dystrophy and MLD to Newborn Screening Recommendations

Defense Bill Orders GAO Lyme Origins Probe as RFK Jr. Pledges Diagnostic Push

Nektar’s Alopecia Drug Misses Phase 2b Goal as Company Cites Ineligible Patients, Plans Phase 3

Sanofi Ends PPMS Bid After Tolebrutinib Fails Phase 3 as FDA Delays Review for nrSPMS

FDA Approves Amgen’s Uplinza for Generalized Myasthenia Gravis

Ianalumab Plus Eltrombopag Extends Disease Control in Phase 3 ITP Study Published in NEJM

New Genomic Study Identifies Chromosome 10 Variants Driving High-Risk AMD Subtype

Shingles Vaccine Linked to Lower Dementia Deaths in Welsh Quasi-Experiment

Largest Sickle Cell Transplant Analysis Finds Durable Survival and Disease Control

UNAIDS Warns Global HIV Response Is in Crisis as Funding Cuts Gut Prevention

Blocking NLRP3 Prevents Early AMD-Like Retinal Damage in Mice, Study Finds

AGA Overhauls Crohn’s Treatment With Living Guideline Favoring Early High‑Efficacy Therapies

Sorfequiline Outperforms Standard TB Regimens in Phase 2 Trial

EMA Panel Backs Higher-Dose Spinraza for 5q SMA

Alzheimer’s Social Memory Loss Linked to Perineuronal Net Breakdown in Mice

Sarepta Shares Plunge After ESSENCE Phase 3 Miss in Duchenne Trial

New Tupac Biography Recounts Janet Jackson HIV Test Request on Poetic Justice Set

Trailer Released for Chris Hemsworth’s Alzheimer’s Documentary ‘A Road Trip to Remember’

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy