Leukodystrophy

After ten years of driving awareness and research around a rare disorder called h-abc/tubb4a related Leukodystrophy it’s become painfully clear a path to a cure for my child was out of reach. She turns 19 this year and although she is healthy her condition is worsening with slow loss of movement. Trials take years and many don’t even make it to finish line. And this is even more prevalent with rare diseases. Then I found Nlorem who creates ASO therapeutics for nano rare conditions. They developed a treatment for her within a year and we are submitting to the fda this Fall for approval. We hope this treatment can be used for the larger population. Nlorem.org.

Michele Sloan
Foundation to Fight H-abc
h-abc.org
#RareDisease
#fighthabc