Nasdaq

PTC Therapeutics Announces Results from Long-Term AADC Deficiency Gene Therapy Treatment Demonstrating Sustained Improvements

SOUTH PLAINFIELD, N.J., Oct. 24, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced new results from its investigational gene therapy, PTC-AADC, in patients living with ...
The Trentonian

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

SOUTH PLAINFIELD, N.J., Aug. 23, 2021 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the publication of a manuscript, “Gene Therapy in the Putamen for Curing AADC Deficiency and ...
Monthly Prescribing Reference

Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval

The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
Nasdaq

New Data Shows Devastating Impact of AADC Deficiency on Caregivers

SOUTH PLAINFIELD, N.J., May 17, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data from the first study on the impact of caring for a child with the ultra-rare genetic ...
New Atlas

Gene therapy success offers hope for reversing rare genetic diseases

A landmark study published in the journal Nature Communications is describing the extraordinarily successful results of a Phase 1 trial testing a targeted gene therapy for children with a rare genetic ...