For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...
Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...
Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant ...
Sinar Daily on MSN6d
Passing on destructive geneMucopolysaccharidosis Type II (MPS II) also known as Hunter Syndrome is when the body lacks the enzyme to break down sugar ...
Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will feature oral and poster presentations at the upcoming ...
REGENXBIO's partnership with Nippon Shinyaku provides $110M upfront, ensuring a cash runway for gene therapy programs ... two gene therapies to treat Mucopolysaccharidosis I & II (MPS).
Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?
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