The latest news from PTC

Emma shares her story about living with PKU and her advice for others.

Pam and her daughter, Kelsey, live with phenylketonuria (PKU), a rare, inherited metabolic disease which affects the brain.

As November ushers in a season of gratitude, it also brings with it National Caregivers Month – a time to recognize and celebrate the unsung heroes who dedicate their lives to caring for others, particularly those in the rare disease community. These individuals, often family members or close friends, play an invaluable role in supporting…

Laci shares what it was like to be a caregiver at a young age for someone with Huntington’s disease.

Londen shares her story as the mother & caregiver of Autumn, who has juvenile Huntington’s disease.

In honor of International Ataxia Awareness Day (IAAD), recognized on September 25th each year, we are joining forces with the global ataxia community in their efforts to bring awareness to this rare disease. The theme of this year’s day is “Ask Me About Ataxia” – encouraging people to learn more about ataxia by asking questions.…

Kailey is grateful for the Friedreich's ataxia community and strives to raise awareness of FA.

Brittany encourages others with Friedreich's ataxia to take their time accepting their diagnosis.

Tomáš wants to remain as independent and active as possible with FA for as long as possible.

PTC research highlights opportunities to improve the transition experience for young adults living with Duchenne In 2023, PTC supported a research project conducted by a patient-focused consultancy Redline Strategic exploring the experience for teenagers, young adults and caregivers living with Duchenne muscular dystrophy (Duchenne) as they transition from pediatric care to adult care. The research…