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MSUD Gene Therapy

Branched-chain α-ketoacid dehydrogenase deficiency (maple syrup urine disease): Treatment, biomarkers, and outcomes

February 18, 2022March 10, 2020 by admin
Categories MSUD Gene Therapy, Publications Leave a comment

Liver Transplantation for Classical Maple Syrup Urine Disease: Long-Term Follow-Up in 37 Patients and Comparative United Network for Organ Sharing Experience

February 18, 2022January 22, 2012 by admin
Categories MSUD Gene Therapy, Publications Leave a comment

Classical maple syrup urine disease and brain development: Principles of management and formula design

February 18, 2022April 26, 2010 by admin
Categories MSUD Gene Therapy, Publications Leave a comment

One Community’s Effort to Control Genetic Disease

February 18, 2022October 11, 2001 by admin
Categories MSUD Gene Therapy, Publications Leave a comment

Recent Posts

  • Director, Gene and Cell Therapy
  • Challenges and opportunities when transitioning
    from in vivo gene replacement to in vivo CRISPR/
    Cas9 therapies – a spotlight on hemophilia
  • New Agreement Set to Scale Up Production of Hem A Gene Therapy
  • Charles River and ASC Therapeutics to Scale Manufacturing of Second-Generation Gene Therapy for Hemophilia A
  • ASC Therapeutics Joins U.S. Consortium of Gene Therapy Partners Including the NIH and FDA to Accelerate Treatments for Rare Diseases

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