The European Medicines Agency (EMA), Spinal muscular atrophy (SMA) Europeand the TREAT-Neuromuscular diseases (NMD) network are holding a one-day workshop to discuss, support and advance the development of therapies for the treatment of spinal muscular atrophy (SMA). SMA Europe and TREAT-NMD network are two non-profit organisations that bring together patients and academics from Europe and beyond to progress research into SMA and facilitate the development of new therapies.
The workshop will take place at EMA’s premises in London on 11 November 2016, starting at 09:00.
SMA is a rare genetic disease that affects the control of muscle movement. It is caused by a loss of specialised nerve cells, called motor neurons, which results in weakness and wasting (atrophy) of muscles used for activities such as crawling, walking, sitting up, and controlling head movement. Children born with the most serious form of the disease rarely survive beyond the age of two because of severe breathing problems.
At present there is no cure for SMA. Available treatments focus on managing symptoms and preventing complications. However, new therapies are currently being developed and SMA might become one of the first inherited neurological diseases to be successfully treated. A number of experimental therapies are currently being evaluated in early clinical trials and EMA has provided scientific advice to medicine developers on the appropriate tests and studies to be used in the development of medicines for SMA a number of times in the past few years.
The workshop will convene key stakeholders – patients, doctors, industry representatives, researchers and regulators – to take stock of the latest scientific developments in the area and discuss ways of developing therapies for the treatment of SMA. Topics for discussion will include an overview of the disease, the pharmacology of the molecules under investigation, natural history data, clinical outcome measures and potential use of biomarkers in drug development.