FDA Grants Orphan Drug Designation to Cytokinetics Therapy CK-2127107 for Spinal Muscle Atrophy
The U.S. Food and Drug Administration (FDA) has granted Cytokinetics’ investigational spinal muscular atrophy (SMA) treatment CK-2127107 Orphan Drug…
Articles by Magdalena Kegel
SMA Genetic Carrier Status Test Being Launched in Europe, Latin America
A new test that can detect the risk of having children with spinal muscular atrophy (SMA) will be commercialized in…
Spinraza Clinical Trial Updates Confirm Treatment Benefits in All SMA Types
A Phase 3 clinical trial evaluating Spinraza (nusinersen) in children with type 2 spinal muscular atrophy (SMA) may play…
Updates From Trial of AveXis SMA1 Gene Therapy Trial Show Impressive Results in Most Infants
Results from the first clinical trial of AVXS-101, a gene therapy for infants with spinal muscular atrophy (SMA) type 1,…
Excessive Levels of Satiety Hormone Leptin May Contribute to Severity of SMA, Study Finds
Nearly half of young people with spinal muscular atrophy (SMA) — and especially those who are underweight — have abnormally…
Children with Spinal Muscular Atrophy Have Poor Bone Health, Study Finds
Children with spinal muscular atrophy (SMA) have poor bone health that leads to common thigh-bone fractures, regardless of the state…
Case of Newborn with Worst Type of Spinal Muscular Atrophy Underscores Gene’s Protective Role
Fetuses with the most severe form of spinal muscular atrophy (SMA) usually die in the womb, but a newborn with…
Orphan Drug Act Helps Pharmaceuticals But Does Little for Patients, Study Says
An analysis of U.S. orphan drug designations and approvals suggests that current incentives, detailed in the Orphan Drug Act (ODA)…
Why SMN Deficiency So Quickly Kills Motor Neurons Detailed in Study with Relevance to SMA Treatment
Researchers may finally have figured out why a loss in the SMN protein that is crucial for all cells so quickly affects…
Manipulating Protein and Gene Seen to Improve SMA Motor Symptoms in Animal Study
Lowering levels of a newly identified protein, in combination with a splice-modifying therapy, improved symptoms and survival in animal models…