News from the H-ABC Community

October 2024 | First Child to be Treated | The first child living with H-abc/Tubb4A was treated in September 2024 using an individualized ASO developed by N-Lorem. Follow our Blog for updates!

9 May 2022 | FDA Patient Listening Session | Six families met with the FDA to share their experience caring for a child living with H-ABC. This session helps the FDA understand what is important to patients, caregivers and advocates. It helps inform regulatory decision-making, provide a starting point to inform early stage research and development, educate the FDA about the disease and helps the patient community to understand the FDA’s mission and work. A summary of the session is available on the FDA website for global public access.

15 October 2021 | SynaptixBio is working to develop the world’s first treatment for TUBB4a leukodystrophy, including H-ABC – a debilitating and potentially life-limiting neurodegenerative condition.

25 April 2021 | USA TODAY | Choosing Hope | An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future.

13 October 2020 | H-ABC.org Press Release | Foundation to Fight H-ABC announces sponsor research agreement with University of Massachusetts in collaboration with Yale University to pursue AAV Gene Therapy for H-ABC.

11 August 2020 | The Sun | MUM'S FIGHT | My girl, 11, can’t walk after a rare disease robbed her of her future – now I’ve helped raise £75k to help find a cure. The Sun profiles the founders of our sister organization in the U.K.

07 July 2020 | New York Times | ‘It Will Consume Your Life’ 4 Families Take On Rare Diseases | The NYT profiles four different approaches to finding treatment for rare diseases.