Medical Xpress

Maple Syrup Urine Disease

A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, ...
IFLScience

Maple Syrup Urine Disease: Why The Formula Shortage Could Also Be Deadly For Some Adults

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.
GEN

AAV Gene Therapy for Maple Syrup Urine Disease Shows Promise

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

Gene therapy prevents death, restores growth in mice and a calf with maple syrup urine disease

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 ...
Business Wire

CORRECTING and REPLACING ASC Therapeutics, UMass Chan Medical School, and the Clinic for Special Children Announce Podium Presentation of Safety and Efficacy in Murine and ...

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

Gene therapy developed for maple syrup urine disease shows promise, new UMass Chan study reports

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 ...
Business Insider

Acer Therapeutics and Relief Therapeutics Announce ACER-001 IND Submission for the Treatment of Maple Syrup Urine Disease

Acer Therapeutics and Relief Therapeutics Announce ACER-001 IND Submission for the Treatment of Maple Syrup Urine Disease 28-Jul-2022 / 07:05 CET/CEST Release of an ad hoc announcement pursuant to Art ...
EurekAlert!

Lipid nanoparticle mRNA therapy improves survival in mouse models of maple syrup urine disease

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 ...
BioWorld

Novel BCKDHB variant and 6q14.1 deletion tied to double diagnosis of maple syrup urine disease and CHUJANS

BioWorld - Tuesday, September 19, 2023 Breaking News: Try BioWorld for free for two weeks See today's BioWorld Science Home » Novel BCKDHB variant and 6q14.1 deletion tied to double diagnosis of maple ...
Nasdaq

Codexis Presents Pre-Clinical Data Highlighting its Programs in Homocystinuria and Maple Syrup Urine Disease at ICIEM 2021

REDWOOD CITY, Calif., Nov. 22, 2021 (GLOBE NEWSWIRE) -- Codexis, Inc. (NASDAQ: CDXS), a leading enzyme engineering company enabling the promise of synthetic biology, today announced that two of the ...
Business Insider

I'm a 28-year-old woman and have maple syrup urine disease. The formula shortage could kill me.

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 ...