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Scholar Rock have presented the latest data from their Phase 2 trial of apitegromab at the 3rd International Scientific Congress on SMA in Barcelona, Spain (October 22nd).

Apitegromab is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by people who live with SMA. In the TOPAZ trial, apitegromab is being tested in combination with SMN-targeted therapies (for example, nusinersen or risdiplam) in people living with SMA Type 2 or 3 and who are unable to walk.

New data after 24 months of treatment indicate that quality of life is either stabilised or continues to improve over the assessment period. This was determined by analysing the effects of SMA through testing abilities to perform daily activities, levels of fatigue, and muscle endurance.

These latest results provide further evidence that apitegromab continues to be safe and well tolerated and support previously reported 24 month findings that the treatment can stabilise or improve motor function (click here for past trial results).

Of the 55 people with SMA who completed the 24 month extension period of the trial, 54 plan to continue to the 36 month stage, when further data will be released.

For additional details on the latest trials results, you can find the Press Release here.
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