The latest news from PTC

Imagine being unable to experience the delight of eating food in a restaurant without fear. For people living with the rare disease familial chylomicronemia syndrome (FCS), this is their unfortunate reality. FCS, which affects one in 700,000 to one million people, prevents the body from breaking down fat, or triglycerides, leading to an accumulation in…

Ethan’s passion for astronomy and physics is “out of this world.”

Jonathan and his entire family’s advocacy efforts are driven by what he calls “tenacious ferocity”.

Kelly Heger became a nurse after her daughter, Jillian, was diagnosed with AADC deficiency.

In this issue of Patient Voices, Jenna shares her journey with HD.

Elizabeth’s journey with Duchenne Muscular Dystrophy (DMD) began when her son was diagnosed at the age of five. In this episode, Elizabeth reflects on the diagnosis of her son, her daughter’s and her own carrier diagnosis, family life, her coping mechanisms and management of the disease. Although her and her family’s lives have been impacted…

Shalom views Duchenne as a companion and source of inner strength.

From global fellowship program to full-time at PTC: Luke's story.

Sorority and team spirit Today, International Women’s Day, we’re sharing the story of a PTC employee that we are proud to have as part of our team: Isabella Prada, Executive Director, Associate General Counsel & Compliance – LATAM and Data Protection Officer LATAM. Isabella has been with us for four years, leading a department made…

Alexander & Tim share the positive effects of sports for the Duchenne community.