The purpose of this organization is to study and seek a cure for all AP-4 HSP disorders. We aim to improve the quality of life for children impacted by AP-4 HSP by accelerating the research …

Cure AP-4 is a non-profit organization dedicated to finding a cure for Hereditary Spastic Paraplegia types 47 (SPG47), 50 (SPG50), 51 (SPG51), and 52 (SPG52)

A team at Boston Children's Hospital, in partnership with Cure AP-4, has developed a translational research program for AP-4-associated hereditary spastic paraplegia (AP-4-HSP), a group of four ultra-rare genetic disorders:

We aim to improve the quality of life for children impacted by AP-4 HSP by accelerating the research for cures and treatments and providing support for patient therapies critical to their...

By generating the most comprehensive AP-4 Hereditary Spastic Paraplegia (AP-4 HSP) Data Collection Program, we can accelerate research and the development of new drugs, devices, or other therapies. Only you hold the key to unlock future discoveries. Already Enrolled?

2022年9月8日 · Founded in 2016 by the families of two newly diagnosed SPG47 patients, Molly Duffy and Robbie Edwards, non-profit foundation Cure AP4’s gene therapy treatment will address the root cause of AP-4 HSP, a rare neurodegenerative disorder, and is intended to be administered as a single dose for the lifetime of the patient.

2023年12月5日 · Partnership will generate clinical data from patients globally to accelerate identifying and developing therapeutics for AP-4 hereditary spastic paraplegias.

Patients must rely on the personal and individualized medical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder.

Dr. Darius Ebrahimi-Fakhari and his team out of the Rosamund Stone Zander Translational Neuroscience Center at Boston Children’s Hospital have identified a set of small molecules that lead to redistribution of ATG9A in patient cells, including skin cells and cultured neurons.

2023年7月21日 · Charles River takes this into account, and provides a full range of gene therapy solutions, including AAV production through the nAAVigation ® platform. This proprietary technology covers the AAV production needs of partners like Cure AP4, from preclinical R&D phase, through early clinical testing, to commercialization. Watch the Webinar