A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.
A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...
New gene therapy for maple syrup urine disease shows promise in preventing deadly symptoms in cows and offers hope for patients.
News Medical on MSN2d
Study demonstrates successful gene therapy for maple syrup urine diseaseA study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...
Pharmaceutical Technology on MSN3d
Inborn errors of metabolism elicit unique challenges for therapy developmentAs Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...
“Simply put, we believe the gene therapy demonstrated in both animal ... a privately held biopharmaceutical company developing in-vivo gene replacement, gene editing and allogeneic cell therapies.
A team of researchers has developed a new gene therapy for children with Dravet syndrome, a rare type of epilepsy in children ...
PHILADELPHIA, March 04, 2025 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases ...
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