Gene Therapy Takes Center Stage at 2019 NORD Summit
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare ... Read more
Updates
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HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access ... Read more -
Equine Therapy for AADC Deficiency
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder characterized by muscle weakness, uncontrollable movements, and developmental delays. Many patients with the ... Read more -
PTC Therapeutics Announces No-cost Program for Diagnosing AADC Deficiency
PTC Therapeutics has launched a program to deliver — at no cost — blood tests that can help diagnose aromatic L-amino acid decarboxylase (AADC) ... Read more -
How Will AADC Deficiency Affect my Child’s Life Expectancy?
As a parent, learning that your child has a rare disease like aromatic L-amino acid decarboxylase (AADC) deficiency can be very difficult. One of the first ... Read more
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Rare Disease Film Festival Highlights Patient and Researcher Unity
Rare disease-themed videos glowed on a large screen before an audience of people in ... -
Tips for Schools That Cater to Children with AADC Deficiency
Aromatic l-amino acid decarboxylase (AADC) deficiency is an inherited disorder that affects the nervous ... -
Gene Therapy Candidate PTC-AADC Sustains Motor, Cognitive, Language Improvements for Up to 5 Years, New Data Show
PTC Therapeutics‘ gene therapy candidate PTC-AADC (formerly AGIL-AADC) provided clinically meaningful and sustained improvements ... -
Speech and Language Delays in AADC
Caring for a child with a developmental disorder such as aromatic L-amino acid decarboxylase ... -
Planning for the Future for Your Child With AADC
Being the parent of someone with a chronic disease such as aromatic L-amino acid ... -
Warning Signs of Depression in AADC Deficiency Caregivers
Being the parent or caregiver of a patient with a chronic disease such as ...
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