News Related to Lysosomal Storage Disorders

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Polaryx Therapeutics Advances Phase 2 Trial for Rare Pediatric Lysosomal Storage Disorders

Mar 03, 2026

Polaryx Therapeutics has selected a contract research organization to conduct SOTERIA, a phase 2 trial evaluating PLX-200 across four rare lysosomal storage disorders, with trial initiation planned for the first half of 2026.

India's Rare Disease Patients Face Treatment Halt as Rs 50 Lakh Aid Cap Exhausted

Mar 02, 2026

Over 100 children with rare diseases in India have exhausted the Rs 50 lakh financial assistance limit under the National Policy for Rare Diseases 2021, forcing treatment halts and prompting urgent appeals for continued support.