Drugs Currently Being Tested in Clinical Trials
Drugs Currently Being Tested in Clinical Trials
If you’re interested in you / your child taking part in a clinical trial, your specialist team are the best people to speak to and will know if any you / your child might be eligible for are recruiting in the UK. Please be aware, however, that clinical trials often recruit globally with a very limited number of places.
Some of the drugs currently being tested in clinical trials are ones being tested as ‘combination therapies’. The currently available SMA therapies (Spinraza, Zolgensma and Evrysdi) aim to increase the amount of survival motor neuron (SMN) in the body (referred to as ‘SMN-targeted therapies’). The combination of an SMN-targeted therapy with a second approach targeting muscles may result in a complementary or added benefit. The idea is that this combinatorial strategy will simultaneously treat the underlying cause of SMA, as well as the symptoms of the condition.
SRK-015 / Apitegromab is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. The drug is an antibody that very specifically targets an important protein called myostatin. Myostatin is naturally present in our muscles, where it plays an important role in limiting muscle growth. By inhibiting myostatin function, Apitegromab can potentially release the breaks on muscle cell growth leading to an increase in muscle size and function. If this is successful, this could benefit people who have SMA.
Read more about trials and any updates, including information about access in the UK >
GYM329 is an investigational anti-myostatin antibody that is designed to target skeletal muscles (i.e., those used for voluntary movement), potentially increasing their size and growth. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. GYM329 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and outcomes for people living with SMA Type 2.
Read more, including information about proposed access in the UK >
Taldefgrobep alfa is a protein designed to bind to and inhibit the function of myostatin. Myostatin is naturally present in our muscles, where it plays an important role in limiting muscle growth. By inhibiting myostatin function, taldefgrobep alfa can potentially release the breaks on muscle cell growth leading to an increase in muscle size and function. If this is successful, this could benefit people who have SMA.
Read more, including information about proposed access in the UK >