Author Archives: Malika Ammam, PhD

SMA Patients Being Recruited for Phase 2 Study of Skeletal Muscle Therapy

A Phase 2 clinical trial investigating a potential treatment, CK-2127107, in people with spinal muscular atrophy (SMA), ages 12 years and older, is currently recruiting patients in the U.S. with varying degrees of disease severity (Types 2, 3, or 4). CK-2127107 is an investigational drug candidate designed for skeletal muscle and other debilitating neuromuscular…

Genetic Therapy Restores Motor Neurons in SMA Model

Researchers from The Jackson Laboratory in Maine, in collaboration with Isis Pharmaceuticals, tested a novel therapeutic candidate (antisense oligonucleotides) in genetically engineered mice with spinal muscular atrophy (SMA), observing it could restore some functionality of motor neurons. The study entitled “Systemic, postsymptomatic antisense oligonucleotide rescues motor…