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Experimental Gene Therapy Changes the Life of Polish Schoolgirl With Unusual Brain Condition

A Polish girl born with AADC deficiency, a rare genetic condition, had experimental brain surgery and is recovering well.

Targeting the Putamen with Gene Therapy Leads to Sustained Improvements in Motor and Non-Motor Functions in Children with AADC Deficiency

Targeting the Putamen with Gene Therapy Leads to Sustained Improvements in Motor and Non-Motor Functions in Children with AADC Deficiency
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PTC Therapeutics Launches the PTC Pinpoint Direct™ Program for Patient-Initiated Genetic Testing

Share this article Share this article SOUTH PLAINFIELD, N.J., April 14, 2021 /PRNewswire/  PTC Therapeutics, Inc. (NASDAQ: PTCT) today introduced a new no-charge, patient-initiated testing program in the United States for individuals who have symptoms consistent with, or a diagnosis of, cerebral palsy (CP) with no evidence of brain injury. CP is a group of disorders that affect a person s ability to move and maintain balance and posture. Many of the common symptoms of CP are similar to other neurotransmitter disorders, such as Aromatic L-amino Acid Decarboxylase (AADC) deficiency, resulting in potential misdiagnosis. The program, PTC Pinpoint Direct™-CP Spectrum, is an innovative, genetic testing program which includes expert genetic counseling at no charge for eligible patients.

12 Best Genomic Stocks to Buy Now - Insider Monkey

12 Best Genomic Stocks to Buy Now The World Health Organization defines genomics as the study of genes and their functions and related techniques. Some may confuse genomics with genetics, the main difference between the two is that genetics focuses on a single gene where as genomics addresses all genes and their relationship with each other. COVID-19 virus made the power of genomics more visible. Rene Hendriksen, Professor, and Researcher at the Technical University of Denmark mentioned, “You can see if the continent has been infected with different clones [mutations of COVID-19] or the same clone and then track those to the point of entry,”

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