Polaryx Therapeutics reaffirms its commitment to patients with rare pediatric lysosomal storage disorders on Rare Disease Day. The company emphasizes the urgent need for new treatments, highlighting that 70% of rare diseases begin in childhood.
PARAMUS, NJ, Feb. 27, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel,... The National Organization for Rare Diseases (NORD), a rare disease patient advocacy organization, notes that a disease is considered rare when it affects fewer than 1 in 2,000 people globally, with approximately 70% of these diseases starting in childhood. There are more than 6,000 rare diseases identified, 72% of which are genetically inherited. “On Rare Disease Day, Polaryx proudly stands alongside the rare disease community - patients, families, caregivers, researchers, and advocacy partners to underscore the urgent need for new and transformative treatments,” said Alex Yang, Chairman and Chief Executive Officer of Polaryx Therapeutics, Inc. For more information about Rare Disease Day, please visit www.rarediseaseday.org. ... SOTERIA is a Phase 2, open-label, single arm trial intended to assess the safety, tolerability, and clinical activity of Polaryx’s lead drug candidate, PLX-200, in CLN2, CLN3, Krabbe disease, and Sandhoff disease, four different LSDs whose patient populations Polaryx believes represent approximately one quarter of the LSD population. A natural history study is a preplanned observational study intended to track the course of the disease. Should the data demonstrate compelling clinical activity, Polaryx may seek conditional marketing authorization. ... Polaryx Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing patient-friendly small molecule and gene therapy treatments for rare orphan lysosomal storage disorders (LSDs).
Original title: “Polaryx Therapeutics Marks Rare Disease Day, Reaffirming Commitment to Patients with Rare Pediatric Lysosomal Storage Disorders”