Eurordis has welcomed as a “significant stride forward” the latest European Parliament plenary vote for compromise amendments to bridge the gap between accelerated scientific progress and patient care. The recent vote was in favor of proposed reforms to the European Union’s general pharmaceutical legislation, which includes proposed…
Read more
Our daughter, Rylae-Ann, failed to meet her milestones at 3 months old. From that point on, she was limp and barely moving. However, she was still very much full of life, and my wife, Judy, and I would help her to still experience the world. Even before we learned Rylae-Ann…
The World Orphan Drug Alliance, known as WODA — a global coalition of pharmaceutical partners with rare disease portfolios — is expanding to cover more countries in Asia and Africa, according to a press release from the organization. The overarching aim, according to the alliance, is to provide greater…
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.
Get regular updates to your inbox.
