For months, those within the rare disease community have been watching to see whether Upstaza (eladocagene exuparvovec) would be approved for use within the European Union. In fact, in…
Kelly Heger, founder of the AADC Family Network, shares her story of becoming a nurse to take care of her daughter with AADC deficiency. When my daughter, Jillian, was born…
PTC Therapeutics Inc. has just announced that their gene therapy called Upstaza has received a positive opinion from the EMA. After the therapy is ratified by the European Commission (EC),…
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…
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.…
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…
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…
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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