With the data of 530 patients from 28 institutions across 15 states, access to researchers to develop treatments adds to the existing knowledge base for the rarest of diseases ...

Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (“LSDs”), announces it will be ...

The SOTERIA trial represents a flexible and resource‑efficient mechanism to continue the clinical development of PLX‑200 across multiple LSDs. Polaryx received a safe to proceed letter from the FDA in ...

HYOGO, Japan, February 05, 2026--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic ...

Ahmedabad: The city recently got the national biobank for lysosomal storage disorders at the FRIGE Institute of Human ...

Oral presentation highlighted the effect of gemfibrozil on Krabbe disease, one of four LSDs in the upcoming Phase 2 SOTERIA basket trial evaluating PLX-200 (reformulated gemfibrozil)PARAMUS, NJ, Feb.

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today ...

Researchers at The Hospital for Sick Children (SickKids) have found that inflammation in an immune cell may be responsible in part for some severe symptoms in a group of rare genetic conditions called ...

Polaryx Therapeutics (“Polaryx” or the “Company”) a clinical-stage biotechnology company dedicated to the discovery, development, and commercialization of novel, disease-modifying therapies for rare, ...