Amyotrophic lateral sclerosis, or ALS, is a progressive neurodegenerative disease that leads to the death of nerve cells that control movement, leaving people unable to move and, eventually, to breathe.
New data have shown for the first time that a combination of targeted therapies can improve survival in patients with advanced bowel cancer. Results of the BEACON CRC Phase III trial have shown that triple therapy targeting BRAF mutations in progressive metastatic colorectal tumors significantly improved overall survival and objective response compared to standard care.
An experimental gene therapy for spinal muscular atrophy (SMA) developed by Novartis AG would be worth up to $900,000, according to an independent US nonprofit organization that reviews the value of drugs and medical treatments.
Biogen Inc.’s Spinraza treatment for spinal muscular atrophy and Swiss drugmaker Novartis AG’s experimental gene therapy are both expensive, but the gene therapy could be more cost effective once more is known about its US price and long-term success rates, a preliminary report from an independent US nonprofit organization said.