Angelman syndrome begins at birth and although symptoms vary, most children exhibit common developmental delays such as: • Infants showing an inability to support their head • Unable to pull…
In the United States, Orphan Drug designation comes with benefits and incentives such as fee waivers, tax credits, and up to seven years of market exclusivity if/when the drug is…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
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