Make Nusinersen available to terminally ill children with Spinal MuscularAtrophy

SMA causes increasing muscle weakness & paralysis. In the most cases babies never sit unsupported, crawl or walk & 95% have a life expectancy less than 18 months as they lose their ability to breathe. Nusinersen has not had 1 side effect in over 4 years of testing. Give SMA babies a chance at life!

SMA is a cruel diagnosis. Knowing there's a safe and well tolerated drug not available is hard to bear. Please read more at www.thefastmovement.org . This drug is showing fantastic results & meaning children never expected to achieve milestones are achieving them for the first time! It is keeping them alive! Early intervention is vital to those with SMA so more of their movement abilities can be saved. With no alternative drugs available, children with SMA can't wait any longer for treatment!

We are committed to ensuring that NHS patients with rare conditions are able to get access to the latest effective medicines on terms that represent value to the taxpayer once they have demonstrated they are safe and of sufficient efficacy and quality.

Spinal Muscular Atrophy (SMA) is a genetic disease that causes muscle weakness and progressive loss of movement. In its most severe form, respiratory problems mean children rarely survive beyond two years of age.

Treatment and support, including exercise and physiotherapy, the provision of assistive equipment and support with nutrition, feeding and breathing is available to help manage the symptoms of SMA and provide the best possible quality of life.

Nusinersen (also known under its developmental names ISIS SMNRx and ISIS 396443), is a medicine being developed by the pharmaceutical company Ionis Pharmaceuticals Incorporated. It is currently undergoing Phase III clinical trials in patients with SMA. Patients may access medicines that are still in development by enrolling in clinical trials for that condition. Details for such studies can be found through the UK Clinical Trials Gateway (www.ukctg.nihr.ac.uk/) by searching for “spinal muscular dystrophy”. Participation in a clinical trial should always be discussed and coordinated with the patient’s doctor.

In order for the company to supply or sell Nusinersen as a medicine, it would be necessary to obtain a marketing authorisation (or licence) which requires the submission of evidence to regulators to demonstrate that the product is safe, has efficacy in the purpose claimed for it, is prepared to the necessary quality standards and that there is a positive balance of benefits over any risks. The company will be able to submit an application to the relevant medicines regulator for evaluation when the clinical trials are completed.