Biomarkers for drug development in propionic and methylmalonic acidemias-Reference-Cited by-同舟云学术

Biomarkers for drug development in propionic and methylmalonic acidemias

Published:2022-01-26 Issue:2 Volume:45 Page:132-143
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J of Inher Metab Disea

Author:

Longo Nicola¹,Sass Jörn Oliver²,Jurecka Agnieszka³,Vockley Jerry⁴

Affiliation:

1. Department of Pediatrics University of Utah Salt Lake City Utah USA

2. Research Group Inborn Errors of Metabolism, Department of Natural Sciences & Institute for Functional Gene Analytics (IFGA) Bonn‐Rhein‐Sieg University of Applied Sciences Rheinbach Germany

3. CoA Therapeutics Palo Alto California USA

4. Division Medical Genetics, Department of Pediatrics University of Pittsburgh, School of Medicine, Center for Rare Disease Therapy, UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania USA

Funder

National Institutes of Health

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jimd.12478

Reference70 articles.

1. Practical management of organic acidemias

2. Guidelines for the diagnosis and management of methylmalonic acidaemia and propionic acidaemia: First revision

3. Inborn errors of metabolism identified via newborn screening: Ten-year incidence data and costs of nutritional interventions for research agenda planning