… the establishment of a national rare disease registry under the health ministry … 45 million people suffering from rare diseases. Malaysia is no exception – around … yesterday called for a national rare disease registry that would consolidate fragmented …

… diagnosis and supporting patients. Since Rare Disease Day launched in 2008, … plausible mechanism approval pathway for rare diseases, intended to expedite treatments “ … not feasible.” This followed the Rare Disease Evidence Principles framework in September …

… , the development of a national rare disease registry, sustainable treatment financing, and … approaches. He also reiterated that rare diseases must no longer be marginalised … a justification to deny treatment.  “Rare diseases must be elevated to become …

… are currently receiving treatment for rare diseases at government hospitals, involving … additional RM5 million allocations for rare diseases, while stressing that he … are strengthening clinical governance for rare diseases, improving early and accurate …

… the National Policy for Rare Diseases, a reform initiative aimed … healthcare ecosystem in addressing rare diseases in a more structured … conjunction with the National Rare Disease Day 2026 observance … nearly 500 types of rare diseases in Malaysia are currently …

… more diverse portfolio of several rare disease therapies. The company acquired … product portfolio, which included the rare disease medications Cholbam and Chenodal. Peetz … . For instance, California-based rare disease biotech BioMarin, which already has …

(WISH) — Rare diseases are individually uncommon but collectively … reminder for families facing a rare disease, answers can take years, and …

… have facilitated a successful rare disease drug pipeline, most rare diseases still do not … a single rare disease or condition and approved for that rare disease or … Act has fundamentally reshaped the rare disease landscape—fueling scientific innovation, …

… of enduring the procedure. MORE RARE DISEASE RECOVERIES: UPDATE Long-Term Follow …

… to add seven more rare diseases for the purposes of … Apart from the drugs for rare diseases, the budget proposal includes … of high-cost treatment of rare diseases. “It does provide significant … customs duty exemption for rare disease drugs is only for …