News

A broad range of clinical studies into AveXis’s gene therapy AVXS-101 is likely to decide whether it is indeed a “transformative” treatment for babies and children with spinal muscular atrophy (SMA) types 1-3. Five trials — ongoing or soon to start worldwide — are planned, including a study in…

The Canadian Institutes for Health Research, a federal government agency based in Ottawa, has awarded genetics professor Kessen Patten C$627,300 to study the mechanisms involved in spinal muscular atrophy (SMA). The leading genetic cause of infant mortality, SMA is a neuromuscular disease that involves the death of lower motor neurons — the…

David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine cancer (PNET) — the same disease that killed Apple’s founder and CEO Steve Jobs. After three years of progress…

Researchers have uncovered the gene network regulating the transition of progenitor cells into motor neurons during the development of embryos, using chicken and mice as models. The study also sheds light on why these neurons develop faster in the embryo compared to other nerve cells. The findings may help researchers…

Scholar Rock’s SRK-015 prevented additional atrophy in mice with muscle wasting and increased healthy animals’ muscle mass and function, a study reports. The biotech company’s therapy targets the precursor to the growth factor myostatin, whose over-activation is linked to muscle atrophy. The study’s findings support SRK-015’s potential as a treatment for muscle…

The modified viral vector being used by AveXis in a range of new or upcoming clinical trials in babies and children with spinal muscular atrophy (SMA) is safe and effective — with distinct differences from the vector used in a recent mammal study that warned of toxicity, company executives…

Early interim data from a Phase 2 clinical trial evaluating varying doses of a potential oral therapy, RG7916, in infants with type 1 spinal muscular atrophy (SMA) is showing good safety and tolerability, researchers said in a recent scientific presentation. Preliminary findings of 13 babies enrolled…

High doses of a modified, non-infectious virus that is being used in gene therapy — including one now in clinical trials in spinal muscular atrophy patients — cause life-threatening toxicity in monkeys and piglets, researchers at the University of Pennsylvania Perelman School of Medicine report. Their study, “Severe toxicity…

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

Infants with spinal muscular atrophy (SMA) continue to show better motor control and strength the longer they receive Spinraza (nusinersen), concludes a new and long-term analysis of final data from a Phase 3 clinical trial in babies with type 1 disease. These infants are also less likely to be…