News

Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?
Experts believe there are only five known cases in the United States, of Beta-mannosidosis, an ultra-rare genetic disorder.
From the time we are conceived and through old age, genetic mutations accumulate in all our tissues, eluding the body's ...
Senators made the right decision for America's most vulnerable patients ― now, the House must follow suit. The ORPHAN Cures ...
Scientists at The Jackson Laboratory (JAX) have developed mouse models that survive premature death and enable pre-clinical ...