- In December 2024, BiolineRx Ltd. (Israel) announced the progress of its clinical trials for a new gene therapy designed to treat Cartilage Hair Hypoplasia (CHH). This groundbreaking therapy, which targets the underlying genetic mutations causing CHH, is expected to provide a novel treatment option to manage the condition’s symptoms and complications. The therapy is currently in Phase 2 trials and has shown promising results in reducing immunodeficiency and improving growth parameters in early-stage patients
- In October 2024, Amgen Inc. (U.S.) revealed new research findings at the Annual Pediatric Genetics Symposium. The study demonstrated the efficacy of a combined therapeutic regimen involving genetic interventions and immunomodulatory drugs in treating patients with Cartilage Hair Hypoplasia. The therapy, currently undergoing clinical evaluation, is expected to reduce the frequency of infections and improve the growth outcomes for children affected by CHH
- In September 2024, Glenmark Pharmaceuticals Limited (India) unveiled an experimental treatment for Cartilage Hair Hypoplasia in collaboration with leading medical research institutions. The innovative drug is designed to tackle the immunodeficiency associated with CHH, with the goal of improving patients' overall health and quality of life. Clinical trials are expected to begin in the second quarter of 2025
- In August 2024, Zydus Group (India) launched a specialized genetic screening test aimed at early detection of Cartilage Hair Hypoplasia. This screening test is designed to identify infants at risk for CHH, allowing for earlier interventions and better management of the condition. The test has been approved for use in hospitals and specialty clinics across India and is expected to be rolled out in other regions in the coming year
