Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening ... Read more
Updates
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Sleeping Tips for Children With AADC Deficiency
Caring for a child with aromatic L-amino acid decarboxylase (AADC) deficiency comes with many challenges. One of the symptoms of AADC deficiency is sleep ... Read more -
2 New DDC Gene Mutations, Identified in African American Siblings, Described in Case Report
Two previously unknown gene mutations that cause aromatic L-amino acid decarboxylase (AADC) deficiency have been identified in two siblings whose cases were described in ... Read more -
Aquatic Therapy for Children with AADC Deficiency
Children with aromatic L-amino acid decarboxylase (AADC) deficiency generally have muscle weakness, uncontrollable movements, and developmental delays. Physiotherapy can help patients with AADC deficiency ... Read more -
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. ... Read more
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Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day ... -
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan ... -
Tips for Feeding Problems in Infants with AADC Deficiency
Caring for a baby with aromatic l-amino acid decarboxylase (AADC) deficiency can be very ... -
Dantrolene Can Prevent Severe Muscle Problems in Children With AADC Deficiency, Case Report Shows
Dantrolene can prevent the destruction of damaged muscle tissue associated with crises of widespread ... -
Study Describes Clinical Signs of 17 AADC Deficiency Patients in Mainland China
A range of disease presentations, and of underlying genotypes, were observed in a study ... -
Physiotherapy for AADC Deficiency – A Parent’s Guide
Physiotherapy is among the treatment approaches likely to help people with aromatic L-amino acid ...
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