Publication: Amylyx Pharmaceuticals, Inc. | Publication Date: April 13, 2023 Authors: Amylyx Pharmaceuticals, Inc. Significance Recently published preclinical data demonstrate initial proof-of-concept for the therapeutic development of AMX0035 (sodium phenylbutyrate and taurursodiol) in Wolfram syndrome Read the entire research article here
Wolfram Syndrome Poster Presentation by Nila Palaniappan-Washington University School of Medicine-Urano Lab
https://www.healthline.com/health-news/how-crispr-gene-editing-may-help-reverse-vision-loss
Wolfram Syndrome Poster Presentation & Abstract by Rie Asada-Washington University School of Medicine-Urano Lab Wolfram syndrome is a rare genetic disorder largely caused by pathogenic variants in the WFS1 gene and manifested by diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration. Recent genetic and clinical findings have revealed Wolfram syndrome as a spectrum disorder. Therefore, a genotype-phenotype […]
RARE DISEASE DAY @ Washington University School of Medicine- Wolfram Syndrome Poster Presentation by Pat Gibilisco and Raquel Gebel-The Snow Foundation for Wolfram Syndrome Research
Late Diagnosis of Wolfram Syndrome, listen to Cathy Gildenhorn’s Wolfram Syndrome story onthe “IT HAPPENED TO ME PODCAST” https://www.ithappenedtomepod.com/episodes/cathy-interview-wolfram-syndrome
Pancreatic Beta Cell May Point to Diabetes Development
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