AADC News

January 14, 2021January 14, 2021

PTC Preparing to Launch Gene Therapy, Pending Approval

AADC News

PTC Therapeutics is preparing to launch PTC-AADC, its experimental gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency, the company has announced in a press release. Among other launch efforts, ... Read more

January 13, 2021January 13, 2021

Paramedical Treatments for AADC Deficiency Patients

AADC News

Paramedical treatment is an important aspect in the care of many chronic diseases, including aromatic l-amino acid decarboxylase (AADC) deficiency. Here is a look at some of the different areas ... Read more

January 7, 2021January 7, 2021

Becoming Teens and Adults With AADC Deficiency

AADC News

Children with aromatic l-amino acid decarboxylase (AADC) deficiency can live to be teenagers and adults, a transition that may lead to changes in disease symptoms and their severity. Care given ... Read more

December 18, 2020December 18, 2020

AADC Deficiency and Ethnicity

AADC News

Aromatic l-amino acid decarboxylase (AADC) deficiency is a very rare disease caused by mutations in the gene that encodes for the AADC protein. The disease seems to be more common ... Read more

December 10, 2020December 11, 2020

PTC Therapeutics Develops Tools to Assess Health-related Quality-of-life

AADC News

PTC Therapeutics has developed and validated tools to help assess health-related quality of life (HRQoL) in people with aromatic l-amino acid decarboxylase (AADC) deficiency. In addition, these health tools are ... Read more

December 9, 2020December 9, 2020

Your Multidisciplinary Treatment Team: Who Will See Your Child With AADC Deficiency?

AADC News

While aromatic l-amino acid decarboxylase (AADC) deficiency primarily affects the nervous system, it also can impact several other systems in the body. Thus, your child will need to see a ... Read more

December 3, 2020December 3, 2020

Centogene, PTC Expand Partnership to Offer AADC Deficiency Testing to More People

AADC News

Centogene has expanded its partnership with PTC Therapeutics to make its genetic and biomarker testing services — to help diagnose aromatic L-amino acid decarboxylase (AADC) deficiency — available free of ... Read more

December 2, 2020December 2, 2020

AADC Deficiency and the Heart

AADC News

As a very rare genetic medical condition affecting the nervous system,  aromatic l-amino acid decarboxylase (AADC) deficiency can have a wide range of symptoms, including movement disorders, oculogyric crises and, in rare ... Read more

November 25, 2020November 25, 2020

Respite Care for Children With AADC Deficiency

AADC News

Providing constant care and supervision to a child who has a chronic disease such as aromatic l-amino acid decarboxylase (AADC) deficiency can be mentally, physically, and emotionally draining. Respite care ... Read more

November 19, 2020November 19, 2020

Trials Data Predict PTC-AADC Gene Therapy to Result in Long-term Benefits

AADC News

PTC Therapeutics’ investigational gene therapy PTC-AADC is expected to result in long-term and durable therapeutic benefits in children with aromatic l-amino acid decarboxylase (AADC) deficiency, long-term data from three clinical trials ... Read more

November 12, 2020November 13, 2020

Scientists Discover 4 Potential Protein Biomarkers for AADC, Other Disorders

AADC News

Scientists discovered four potential protein biomarkers — apolipoprotein D, apolipoprotein H, oligodendrocyte myelin glycoprotein, and collagen6A3 — that may be useful for assessing the severity of certain disorders, including aromatic l-amino ... Read more

November 11, 2020November 11, 2020

Medical Alert Cards for AADC Deficiency

AADC News

Aromatic l-amino acid decarboxylase (AADC) deficiency can cause sudden cardiac issues, oculogyric crises, and problems with breathing and blood pressure, among other symptoms. Physicians may not be familiar enough with AADC to ... Read more

November 4, 2020November 4, 2020

My Child Needs a Lumbar Puncture, Now What?

AADC News

If doctors suspect your child might have aromatic l-amino acid decarboxylase (AADC) deficiency, they may order a lumbar puncture to aid in a diagnosis. Here are some tips on what to ... Read more

October 29, 2020October 29, 2020

Gene Therapy for AADC Deficiency Given Boost by NIH Grant

AADC News

The development of new gene therapy to treat people with aromatic L-amino acid decarboxylase (AADC) deficiency has been boosted by an R21 grant awarded by the National Institutes of Health ... Read more

October 28, 2020October 28, 2020

Feeding Tubes for AADC Deficiency

AADC News

Nursing and swallowing can be difficult for children with aromatic l-amino acid decarboxylase (AADC) deficiency, and raise the possibility of both poor nutrition and choking. Because of feeding problems, these children ... Read more