Foundation to Fight H-ABC BLOG
What a Groovy Fundraiser!
We are so excited to share that our fundraiser in February was an amazing hit! We raised approximately $50,000!! What an amazing accomplishment and we could not do it without the amazing talent of our fundraising team Bob and Mickie Ziarko, their family and friends, the amazing H-abc families who joined us on stage, and The Village people! Thank you everyone for your incredible support!
See LINK to the news media coverage!
January 13, 2024 ~ Drug Repurposing
The Foundation to Fight H-abc is recruiting patients living with confirmed diagnosis of H-abc/Tubb4a related Leukodystrophy for a study for drug repurposing.
The process of drug repurposing uses AI to identify the most promising uses for a specific disease using existing drugs. Then, for our study, the top matches are tested against patient RNA to determine what is a viable candidate. Machine learning is very well-suited to identifying these patterns, which a researcher would not be able to at the same magnitude. Because generic, FDA-approved drugs have a known safety profile and are already manufactured and available worldwide, often with substantial data already existing to support the potential new use, drugs can go through an accelerated research process. These drugs can get to patients quickly, in months versus decades, at a low cost of less than 1% of new drug development.
Go to our Resource/Drug Repurposing page for more information and sign up for this study: https://www.h-abc.org/drug-repurposing
Wishing Connor well!
In our blog in August we shared that there was a treatment available developed by Nlorem, and the first child was to be treated. Connor has now had three doses and doing well. Join us in supporting the family and helping with their costs as they pave the way for potential new treatment for others waiting.
Meet Ben - Living Joyfully with Purpose!
Ben Rosenberg, a vibrant 28-year-old with an unbreakable spirit, was diagnosed in the summer of 2024 with TUBB4a-related Leukodystrophy after a lifetime of uncertainty. This rare neurological condition affects his movement, speech, and balance, but Ben embraces life with a philosophy that inspires everyone around him: "Stay joyful and do the best you can with what you got."