The Medicines and Healthcare products Regulatory Agency (MHRA) is seeking public feedback on a proposal to revamp how rare disease treatments are developed in the U.K. The regulatory body wants input on a new plan that would streamline the clinical testing and approval processes to help get therapies to patients…
Discussion
People often tell me they cannot believe everything my wife, Judy, and I have managed to juggle over the years. They ask how we keep up with the appointments, therapies, advocacy work, and daily responsibilities. The funny thing is that most days we do not even notice it anymore. Life…
EveryLife Foundation for Rare Diseases’ 15th Scientific Workshop recently brought together leaders from the rare disease patient, scientific, government, and biopharmaceutical communities to discuss practical, patient-centered solutions for developing therapies for rare diseases amid evolving FDA pathways and programs. Titled, “Making What Matters Count: Advancing…
Aromatic l-amino acid decarboxylase (AADC) deficiency is a genetic and neurometabolic disease that affects the nervous system. Learn more about the disease here.
AADC deficiency can affect many aspects of a person’s life, including mobility, breathing, sleeping, and eating. Learn more here about different ways to manage the condition, adapt, and handle everyday tasks.
There is not yet a cure for AADC deficiency, but there are approved treatments that can help manage the condition, as well as experimental treatments in the pipeline. Learn more about treating the disease here.
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