A UConn Health Team Administers the World’s First Gene Therapy for Glycogen Storage Disease Type-1a
In January of this year, a University of Connecticut (UConn) team administered the world’s first infusion of a new gene therapy as part of a
The CureGSD1b Research Alliance
The CureGSD1b Research Alliance
CureGSD1b is a group of parent advocates and scientific advisors working together to bring awareness, better treatment, and ultimately a cure to all patients with Glycogen Storage Disease Type 1b. The organization was started in 2020 by the parents of Sophie, who had recently been diagnosed with GSD1b in the fall of 2019. She inspired her parents to create the Sophie’s Hope Foundation, which later led to the formation of the CureGSD1b Research Alliance. Our goal is to create a global network of GSD1b patients, advocates, doctors, and researchers to help patients today, while pushing towards a cure in the future.
Condition Awareness & Advocacy
Here is a list of conditions this partner raises awareness and advocacy for:
Patient Worthy Posts on GSD1b
A Glycogen Storage Disease Patient Story: Fighting for Answers and Finding Community
Written By Katie Ward I am not one who likes to bring attention to themselves, and you won’t ever find me talking about my struggles,
Editor’s Choice: Duchenne, GSD and Orphan Drugs
Happy Friday! This week we have details about an investigative therapy for Duchenne Muscular Dystrophy that has entered a phase 3 clinical trial. Also, have
Rare Classroom: Glycogen Storage Disease Type IB
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on
Life as a Parent of a Child With Glycogen Storage Disease: A Rare Disease Story From the Czech Republic
When your child is constantly at risk of hypoglycemia and their health and life is threatened in the case of any major problem, you have
