Most people with rare diseases given an experimental therapy through compassionate use programs show clinical benefits, and most of these treatments ultimately end up getting approved, according to a review study. Based on these findings, the researchers argued that such programs — allowing certain patients with limited options access to…
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Social networks offer a public platform for people to share their ideas, but authenticity can be a challenge to identify amid the abundant content available. As a parent of a child with a rare and incurable disease, I could be misled by the lofty claims and health trends that show…
About one in 10 medications sold in Canada have a patient support program provided by its manufacturer, and nearly half are for rare diseases. That’s according to a study in Canada that also found that patient support programs were offered more commonly for brand-name and expensive drugs. These programs, offered…
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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