AceLink is advancing a small-molecule treatment option for Fabry disease, which could provide a more convenient alternative ...
LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, ...
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Serious Symptoms Of Fabry's DiseaseFabry's disease is an inherited condition that develops due to a mutation in the GLA gene. For most patients with this disease, the mutation causes the body to produce insufficient amounts of ...
4D Molecular Therapeutics (4DMT) has said a trial of its gene therapy for Fabry disease has been placed on a clinical hold by the FDA, a few weeks after it reported three cases of atypical ...
“We remain focused on advancing our wholly owned neurology genomic medicine pipeline and progressing our Fabry gene therapy program towards a potential BLA submission in the second half of 2025,” ...
Pfizer terminated partnership with Sangamo on hemophilia A gene therapy despite positive Phase 3 data. Find out why SGMO ...
In addition to the Fabry disease program, Sangamo's diverse pipeline includes isaralgagene civaparvovec (ST-920 or isa-vec), a gene therapy program, and STAC-BBB, an innovative approach targeting ...
Sangamo (SGMO) announced updated data from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene ...
In addition to the Fabry disease program, Sangamo's diverse pipeline includes isaralgagene civaparvovec (ST-920 or isa-vec), a gene therapy program, and STAC-BBB, an innovative approach targeting the ...
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