A phase 1b/2a clinical trial to test the safety, tolerability and pharmacokinetics of single and multiple doses of HT-100 (also called delayed-release halofuginone) in boys with Duchenne muscular dystrophy (DMD) is now open at four sites in Maryland, Missouri and Ohio, with an additional site expected to open in California.
Treatment with a small molecule called trehalose was associated with slower disease progression and longer life span in mice with a disease mimicking amyotrophic lateral sclerosis (ALS), an MDA-supported research team has reported.
Trehalose is a compound composed of two glucose (sugar) molecules.
Researchers at Boston University, supported in part by MDA, say their experimental two-pronged strategy for merosin-deficient congenital muscular dystrophy (MDC1A) was highly successful in a mouse model of this disease and should be further investigated as a potential treatment approach for patients.
MDA grantee Matthew Disney is a current and former MDA grantee at the Scripps Research Institute in Jupiter, Fla. Disney's current MDA grant is focused on targeting toxic RNA in type 2 myotonic dystrophy (MMD2, or DM2).