Medical research

Clinical trials & therapy pipeline

There are currently several potential therapeutic products going through both pre-clinical and clinical development. Clinical development, or clinical trial, is a rigorously controlled test designed to examine the safety and/or effectiveness of medicines, devices, treatments, or preventive measures in humans. Clinical trials follow a strict protocol. The protocol describes the aims, design and organisation of a clinical trial. It also provides information on the background and reasons for a trial and outlines its study plan. The plan must be carefully designed to ensure the safety of the participants as well as answer specific research question(s).

Clinical trials for SMA

The identification, development and approval of drugs is a long, costly and difficult process. It is estimated that 9 out of 10 experimental drugs starting human clinical trials never gain final approval.

Researchers have identified four main different therapeutic approaches that show promise in treating SMA (please click on the approaches for more information):

Products in pre-clinical discovery and in clinical trials

therapy pipeline

Clinical trials in the UK and continental Europe

SMN1 gene replacement

  • Avexis AVXS-101Trials are being planned in a few centres across the European Union. Our news items report on recent developments.

SMN2 gene enhancement

  • PTC Therapeutics/ Roche – Moonfish (RG7800) is currently on hold.
  • PTC Therapeutics/ Roche/ SMA Foundation – RG7916 . Recruitment for two phase 2 clinical trials started in December 2016 in SMA types 1, 2 & 3 through trials called Firefish & Sunfish. Recruitment for a 3rd trial, Jewelfish began in 2017.  RG7916 is an orally available drug that aims to correct the splicing of SMN2, the “backup gene.” A community update was provided.

Motor neuron protection

  • Roche (Trophos’) Olesoxime. The company filed an application with the European Medicines Agency in August 2015. The scientific advice received has led to Roche to decide to conduct a phase 3 clinical trial. A community statement was provided.

Clinical trials in the US

SMN2 gene enhancement

  • PTC Therapeutics/ Roche – Moonfish (RG7800) – Patient recruitment currently suspended (see above).

SMN1 gene replacement

Muscle enhancement

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