Rare Disorders NZ believes that precision health provides great opportunity to significantly improve diagnosis, care and treatment, screening, and managing genetics and hereditary issues in families for rare disorders.
Two representatives from the rare disorder community in New Zealand are today participating in a UNESCO-led international meeting to discuss better inclusion in the education system for children living with a rare disorder.
While Rare Disorders NZ will be celebrating this news with the SMA community, frustration remains that it has taken so long to approve this life-changing medicine, having long called for a separate assessment pathway for medicines for rare disorders.
Rare Disorders NZ is pleased to learn that Pharmac’s Pharmacology and Therapeutics Advisory Committee (PTAC) has recommended the life-saving drug Trikafta be funded for eligible people for the age groups 6 years and older and 12 years and older.
Rare Disorders NZ applauds the Government for accepting the Pharmac Review Panel’s recommendation to ‘develop a strategy that will lead to better, more timely services and more equitable support and outcomes for people and whānau with rare disorders’.