Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with sickle ...
Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
This article is part of a Health Affairs Forefront featured topic, Medicaid Financing. Which includes analysis, proposals, and commentary that will inform policies on the state and federal levels to ...
Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV ...
CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...
Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with sickle ...
Regenxbio (RGNX) hit by FDA CRL for RGX-121; valuation discount, cash runway to 2027, risks vs RGX-202 upside. Click here to ...
Hospitals and cancer centers that are launching cell and gene therapy programs need more than scientific breakthroughs to succeed. Delivering these therapies safely and efficientl ...
Regulators overseeing rare disease treatments need better tools to weigh competing risks in real time. Sarepta Therapeutics’ Elevidys is a prime example of why.
Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy treatment appeared at risk. The Food and Drug Administration will request that the company ...
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