Coinciding with Rare Disease Day, Direct Relief is excited to announce a new collaboration with biopharmaceutical company, Shire HGT, to help patients living with lysosomal storage disorders (LSDs) – a class of rare diseases.
In the U.S., a rare disease – also known as an orphan disease – is defined as a disease or condition that affects less than 200,000 people. While the individual conditions are uncommon in themselves, worldwide there are about 350 million people living with one of the estimated 7,000 known rare diseases.
Many rare diseases do not have available treatment, but Shire HGT has developed enzyme replacement therapies (ERT) for Fabry disease, Gaucher disease, and Hunter syndrome – all three are types of lysosomal storage disorders.
To help those living with LSD’s access this treatment and specialty care, Shire HGT and Direct Relief are working to help provide free, high-quality, individualized treatment to patients around the world who would otherwise not be able to receive care.
Direct Relief will serve as a liaison between Shire HGT, the patient and their family, the treating physician, and the medical facility where care and treatment will take place to ensure a regular donation of ERT is delivered and that all medical-related components are in place for treatment to be effectively provided.
This initiative is just beginning, but we look forward to collaborating on behalf of rare disease patients. We will share more about the partnership in the coming weeks.