SMA Press

By Elie Dolgin The most common genetic killer of infants, a disease known as spinal muscular atrophy, is caused by mutations in a single gene. The human genome contains its own backup system—near-identical copies of the defective gene—yet these secondary sequences rarely get used correctly. As Elie Dolgin finds out, drug companies hope to change…

Ongoing Phase 1 Study Shows ISIS-SMNRx Well-Tolerated in Children with SMA CARLSBAD, Calif., November 1, 2012 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced the initiation of a Phase 1b/2a study evaluating ISIS-SMNRx in children with spinal muscular atrophy (SMA).  SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and…

The Economic Power of Orphan Drugs, (Thomson Reuters, August 23, 2012), reveals that today’s blockbuster drugs are not the only money makers by comparing the total projected value of orphan and non-orphan drugs from 1990 to 2030. This analysis is based on a peer-reviewed study from Drug Discovery Today. Following are some of the key…

The Charlie Rose Brain Series explores the study of the human brain. The most recent episode on motor neuron disorders features SMA. Included on the panel of scientists and researchers is Dr. Richard Finkel, Chief of Neurology at Nemours Children’s Hospital and a principal investigator for the SMA Foundation’s Pediatric Neuromuscular Clinical Research (PNCR) Network.…

Drug Firms Engage in Nontraditional Research Partnerships in a Bid to Get Closer to the Patient In the pantheon of companies competing in the health care market, Proteus Biomedical is unique. A start-up based in Redwood City, Calif., Proteus has developed technology to attach a biodegradable digital device to a pill. After a patient swallows…