The American Journal of Managed Care

Real-world Evidence Supports Conditional Use of IV Formula in Hospitalized Infants With MSUD

Hospitalized infants with the rare metabolic disorder maple syrup urine disease (MSUD), who are intolerant to oral or enteral administration of branched-chain amino acid-free formula, may benefit from ...
Business Insider

Relief Therapeutics and Acer Therapeutics Announce that the European Commission Has Granted Orphan Drug Designation for ACER-001 in Maple Syrup Urine Disease

Relief Therapeutics and Acer Therapeutics Announce that the European Commission Has Granted Orphan Drug Designation for ACER-001 in Maple Syrup Urine Disease 12-Aug-2022 / 07:00 CET/CEST Release of an ...
EurekAlert!

Only transplant center that developed comprehensive protocol for liver transplants in kids with MSUD

One year ago, Children's Hospital of Pittsburgh became the first and only transplant center in the world to develop a comprehensive, multidisciplinary medical protocol for performing liver transplants ...
News Medical

30-year study of MSUD highlights critical need for more effective disease-modifying therapies

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 ...
Nasdaq

Orphan designation provides potential for up to 10-year market exclusivity in the EU upon regulatory approval

GENEVA, Switzerland and NEWTON, Mass., Aug. 12, 2022 (GLOBE NEWSWIRE) -- RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief), and its ...
Nasdaq

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

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: ...
AZoNano

Engineering Lipid Nanoparticles for Enhanced MSUD Treatment

A groundbreaking study by researchers at the University of Pennsylvania and Moderna has shown that repeated mRNA therapy can significantly improve survival and reduce leucine levels in a mouse model ...
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 ...
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

ASC Therapeutics Announces Partnership With University of Massachusetts Medical School to Co-Develop Novel Gene Therapy for Maple Syrup Urine Disease

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 ...
Business Insider

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

Every time Heather publishes a story, you’ll get an alert straight to your inbox! Enter your email By clicking “Sign up”, you agree to receive emails from ...