A US FDA panel of advisors concluded that there was a lack of substantial evidence to indicate that Biogen’s investigational drug, tofersen, was effective in treating a rare and aggressive form of ALS. But the experts voted unanimously that the investigational...
Curamys, a South Korean biotech developing cell and gene therapy using cell fusion technology to treat rare intractable diseases, has signed a strategic platform licensing agreement with US tech developer, MaxCyte.
The European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for review of tofersen, an investigational drug for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
The US Food and Drug Administration (FDA) has awarded 19 new grants and two new contracts totaling more than US$38m in funding over the next four years to support clinical trials and natural history studies related to rare diseases.
A neuroprotective drug which is already in development may be particularly effective for MND patients carrying a newly discovered genetic risk factor, helping to halt progression or even prevent the onset of the devastating degenerative disease.