Reading Time: 5 minutes Last Thursday marked an exciting milestone in the search for effective therapies for MND as full results from the early-phase 1/2 trial of Biogen’s tofersen antisense oligonucleotide therapy for SOD1-ALS were published in The New England Journal of Medicine. In the same issue, a study investigating the use of microRNA in a viral vector to…
Reading Time: 2 minutes This article was written by Dr Keith Mayl and Dr Ahmad Al Khleifat of King’s College London. Researchers at King’s College Hospital, led by Professor Christopher Shaw, have embarked on the first gene therapy clinical trial for patients affected by a specific genetic form of ALS, the most common type of MND. ALS is a…
Reading Time: 4 minutes On 22 September 2014 Neuralstem announced the results from their long-term follow up of three participants who were involved in their initial phase I safety trial. Here’s what the news means and what may be next for Neuralstem’s stem cell treatment. What is Neuralstem? Neuralstem is the company behind an innovative neural stem cell treatment for…
Reading Time: 3 minutes Results from a phase I clinical trial of a drug known as ISIS 333611 have been published open-access online in the scientific journal Lancet Neurology on 29 March 2013. This is the first time researchers have tested the effects of delivering an antisense oligonucleotide directly into the human cerebral spinal fluid (the fluid between the spinal…
