A recent discovery is bringing new hope to those suffering from autosomal dominant leukodystrophy (ADLD), a rare and fatal genetic disorder. ADLD is caused by a duplication in the LMNB1 gene, leading to excess levels of the Lamin B1 protein. This buildup damages the protective myelin surrounding nerves in the brain and spinal cord.
While symptoms often don’t appear until the 4th or 5th decade of life, the gradual nerve deterioration is untreatable and ultimately fatal. Patients typically survive for 10-20 years after diagnosis.
In 2019, a small group of patients and families founded the ADLD Center to advocate for research and support. Their goal is to unite the ADLD community and drive progress toward new therapies. The center shares the latest research news and resources for patients in a monthly newsletter.
There are many ways to get involved and accelerate treatments for this devastating disease. The ADLD Center is seeking more patients to join their registry, which signals to drug companies the urgent need for solutions. They also welcome volunteers to fundraise, write for the newsletter, and connect with researchers.
While ADLD remains challenging to treat, the ADLD Center empowers patients to shape the search for a cure. Through grassroots organizing and amplifying patient voices, their efforts bring the hope of finding therapies to slow or stop ADLD’s progression.