lt;pgt;Scientists have successfully reconstructed a virus thousands of years after it became extinct, a development they believe could herald a new step in treating genetic diseases such as cystic ...
In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...
Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene ...
A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...
FOX 11 Los Angeles on MSN
USC professor pursues gene therapy research in quest for an HIV cure
Dr. Paula Cannon is pursuing one of medicine’s most difficult goals: a cure for HIV.
In LAMA2-related muscular dystrophy, weakened extracellular matrix (yellow) leads to muscle fiber degeneration and the infiltration of immune cells (red) into the tissue. Researchers at the University ...
Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big hurdle for patients seeking this specialized care.
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 ...
Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...
Cell and gene therapy (CGT) has evolved from a niche research pursuit into a central force in biopharmaceutical innovation. By engineering living cells and genetic material to repair or replace faulty ...
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.
Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...
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