Additional results of garetosmab in FOP
Compared to a placebo, garetosmab would sustainably reduce not only the frequency of attacks of fibrodysplasia ossificans progressiva (FOP) but also their severity, their median duration (15 versus 48 days) and the need for corticosteroids (40% versus 58 %). These results come from a new analysis of data from the Lumina-1 trial, published while the evaluation of garetosmab continues in particular in France with the phase III trial, Optima, recruitment of which has been completed. As a reminder, this drug candidate blocks the activin A signaling pathways, which are abnormally activated in FOP.
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– Other treatment options for FOP
Subcutaneous efgartigimod injection compared to infusion
The Adapt-SC trial included 110 adults with a generalized form of autoimmune myasthenia gravis. They received efgartigimod (Vyvgart®) either by injection under the skin or by intravenous infusion, once a week for one month. The effect of the subcutaneous route proved to be non-inferior to that of the intravenous route on the main criterion of the study: the reduction in the level of antibodies (IgG) in the blood. The two routes of administration also had similar effectiveness on the manifestations of the disease (MG-ADL score in particular). Published at the same time, the preliminary results of the extension of this trial, called Adapt-SC+, support these results.
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Thiamine, a food supplement to be evaluated in CMT
Thiamine, or vitamin B1, contributes to the proper functioning of the nervous system and its administration could have beneficial effects in neurological diseases such as Parkinson’s disease or Friedreich’s ataxia. A pilot study in 15 patients with Charcot-Marie-Tooth disease showed that daily oral intake improves hand strength and restores certain physiological parameters (nerve conduction speeds). If these preliminary results seem promising, it is necessary to confirm them with a large clinical trial.
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