The Access to Treatment Committee is made up of pharmaceutical executives, physicians, academic professors, patient advocates, and a bioethicist. This committee will review applications from physicians or organizations, like the UDN, to treat diagnosed, genetically confirmed nano-rare disease patients. Applications will be approved and prioritized based on criteria such as: feasibility of developing an ASO treatment for the genetic cause of the disease, degree of potential benefit of treatment, practicality of treatment, availability of physician and institution to treat patient and conduct necessary studies, and other complexities of the condition.
At the Foundation we understand that there are seriously ill patients with immediate needs of treatment for their condition. We are sympathetic to patients and all who are involved. The Committee will fully review all applications received before a decision is made. We plan to do all we can to make sure we are as transparent as possible. Due to the complex nature of treating patients with nano-rare diseases, unfortunately not all can be approved for treatment.