PTC-AADC Shows Benefits for Patients with AADC Deficiency, Data Shows

Recently, biopharmaceutical company PTC Therapeutics, Inc. performed a five-year data analysis to evaluate the continued safety, efficacy, and tolerability of PTC-AADC. The company developed this novel gene therapy to improve…

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Study Illuminates Impact of Aromatic L-Amino Acid Decarboxylase Deficiency on Caregivers
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Study Illuminates Impact of Aromatic L-Amino Acid Decarboxylase Deficiency on Caregivers

PTC Therapeutics has just announced data from the very first study which examined how taking care of a child diagnosed with aromatic L-amino acid decarboxylase (AADC) deficiency can impact caregivers.…

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Expanded Partnership Will Offer Genetic Testing for AADC Deficiency
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Expanded Partnership Will Offer Genetic Testing for AADC Deficiency

  As of the end of August 2020, CENTOGENE has collaborated with more than 40 pharmaceutical partners to develop solutions for patients with rare diseases. In a press release from…

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Dantrolene Effective for Muscle Problems in AADC Deficiency Children: Case Study
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Dantrolene Effective for Muscle Problems in AADC Deficiency Children: Case Study

By Jodee Redmond from In The Cloud Copy The results of a case study have found that dantrolene can be used to treat involuntary muscle contractions (dystonia) in children with…

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Gene Therapy Proves Effective in Treating AADC Deficiency in Trial
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Gene Therapy Proves Effective in Treating AADC Deficiency in Trial

According to the publication over the weekend from San Francisco CBS affiliate KPIX, a groundbreaking clinical trial for those with aromatic l-amino acid decadboxylase (AADC) deficiency was undertaken at UCSF…

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