Maple syrup urine disease (MSUD) is a rare genetic inborn error of metabolism characterized by recurrent life-threatening neurologic crises and progressive brain ...

A one-time injection of a novel gene therapy was able to save mice and a newborn calf from succumbing to maple syrup urine disease (MSUD), suggesting the approach could one day be used to treat ...

IFLScience needs the contact information you provide to us to contact you about our products and services. You may unsubscribe from these communications at any time.

NEWTON, Mass. and GENEVA, Switzerland, July 28, 2022 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (Nasdaq: ACER) (Acer) and its collaboration partner, RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: ...

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 ...

MILPITAS, Calif.--(BUSINESS WIRE)--Please replace the release dated May 16, 2022 with the following corrected version due to multiple revisions. The updated release ...

MILPITAS, Calif.--(BUSINESS WIRE)--ASC Therapeutics, a privately-held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell ...

New Rochelle, NY, August 21, 2024—Researchers from the University of Pennsylvania, Perelman School of Medicine, Gene Therapy Program, and Moderna, have shown that repeated administration of lipid ...

Hannah Dolins has maple syrup urine disease and needs a metabolic formula to live. Without her formula, she can fall into a coma and die. This is her story, as told to Heather Marcoux. This as-told-to ...

Maple syrup urine disease (MSUD) is caused by an inherited deficiency of the branched-chain α-keto acid dehydrogenase, resulting in a marked increase of the branched-chain amino acids (BCAAs), leucine ...

A new study analyzes 30 years of patient data and details the clinical course of 184 individuals with genetically diverse forms of Maple Syrup Urine Disease (MSUD), which is among the most volatile ...