Gene therapy for maple syrup urine disease shows promise
New gene therapy for maple syrup urine disease shows promise in preventing deadly symptoms in cows and offers hope for patients.
Gene therapy developed for maple syrup urine disease shows promise
A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...
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Study demonstrates successful gene therapy for maple syrup urine disease
A 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 ...
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Inborn errors of metabolism elicit unique challenges for therapy development
As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...
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Gene therapy developed for maple syrup urine disease shows promise, new UMass Chan study reports
“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.
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Hope for kids with rare epilepsy
A team of researchers has developed a new gene therapy for children with Dravet syndrome, a rare type of epilepsy in children ...
Why pharma is still far from a treatment for galactosemia
With issues in therapy delivery and a barren trial landscape, short-term chances for a commercial galactosemia treatment are ...
uniQure N.V.: A Potential Huntington's Play
Qure's stock rises on Huntington's therapy progress and strong gene therapy pipeline. Learn about QURE stock's cost-cutting ...