News

Harvard Team Finds Compound to Restore Protein That Is Short in SMA

Researchers at the Harvard Stem Cell Institute have identified a compound that can restore the SMN motor-neuron protein whose shortage is associated with spinal muscular atrophy (SMA). “This discovery opens up new lines of drug interrogation,” Lee Rubin, the senior author of the study, said in a news story written by Harvard’s Hannah…

Cytokinetics Enrolling Second Group of SMA Patients in Phase 2 Trial of CK-2127107

Cytokinetics has begun enrolling a second group of patients for a Phase 2 clinical trial of CK-2127107 as a treatment for spinal muscular atrophy (SMA). The announcement follows an independent Data Monitoring Committee’s review of the therapy’s safety, absorption, distribution, metabolism, and excretion in Cohort 1. The randomized, double-blind, placebo-controlled,…

Invitae Rolls Out New Genetic Test to Diagnose SMA

San Francisco-based Invitae has developed a genetic test to diagnose spinal muscular atrophy (SMA), the company announced at the March 21-25 American College of Medical Genetics (ACMG) Annual Clinical Genetics Meeting in Phoenix. SMA is the second leading genetic disorder affecting children in the United States. One in every 10,000 kids has SMA,…