Page 4 - Inborn Errors News Today

FBX-101 clinical data demonstrating safety and initial efficacy from the RESKUE trial is being presented at the 2022 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium

Isaralgagene civaparvovec, or ST-920, continued to be generally well tolerated across three dose cohorts in the six treated patients The five longest treated patients exhibited elevated a-Gal A activity

Press release content from Globe Newswire. The AP news staff was not involved in its creation.

AUSTIN, Texas, Aug. 29, 2022 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company developing a new ...

/PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to...

The Business Research Company’s Metabolomics Global Market Report 2022: Market Size, Trends And Forecast To 2026

/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium,...

Rise in prevalence of malnutrition, faster response time, and high efficacy associated with intravenous solution therapy have boosted the growth of the global intravenous solutions market. PORTLAND

KEY HIGHLIGHTS Paradigm has been invited to present results from the mucopolysaccharidosis (MPS) program, with an oral presentation at the XVII International...

Here is how late-stage gene therapy programs offer a glimpse into the future of rare disease medicine.