Many of you have been following us now for almost ten years, in our quest to find a cure for Elouise and kids like her living with H-abc/Tubb4a related leukodystrophy. We have been aggressively gaining awareness around this condition and engaging with researchers who are actively working on a cure. In fact two possible treatments are in the works for the condition! However, the process for full clinical trials to create and develop a commercial drug is very slow and we are running out of time! Elouise turned 19 in September and we know if we don't get her access to a treatment, she will deteriorate to the point of dysfunction.
With all the increased awareness around rare diseases and lack of treatments, a non profit called Nlorem (nlorem.org) ventured into developing individual treatments for nano rare conditions that have no cure, processed through the FDA under an individualized accelerated protocol, designed specific to each individual. Nlorem develops and donates therapeutics to the patient for life, and partners with genetic physicians to administer the drug which is administered quarterly. We applied for Elouise, provided cell samples and they have developed a drug for her specific genetic make up! At this time, they are about to submit to the FDA for initial approval to proceed with her study!
However, under this program, until a drug has been fully vetted and approved by the FDA as meeting all safety and outcome protocols, insurance will not pay. Once approved in final by the FDA, insurance is required to pay. The study will take 1-2 years. We are seeking donations to help pay for the hospital costs.
What we are about to embark on has its risks, but if we don't take action, the long term outlook is bleak. But the good news is, the treatment developed by Nlorem could be used for the rest of the kids once proven to be effective. There are other kids on this same path, collectively we can take the drug to the FDA and pursue approval for commercialization and help finish what we set out to do, eradicate H-abc for all the kids!
Elouise is a hero, please join us in making this happen. We are very grateful for your ongoing support!
Britt and Michele Sloan
