Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
Morning Overview on MSN
Gene hacking is coming and it could rewrite what it means to be human
For the first time, the tools to rewrite human biology are moving from speculative fiction into regulated clinics and ...
ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...
A free two-day event in Cambridge will unite experts to discuss translational advances in cell and gene therapy, spanning rare diseases, cancer immunotherapy, and genome editing.
What Is Waskyra, and Why Does It Matter? Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune ...
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 ...
Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...
Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
The company said that the FDA hold has impacted two experimental gene therapies, RGX-111 and RGX-121. ・REGENXBIO added that ...
Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
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