A multisite, international phase 2 trial evaluating the investigational drug garetosmab has shown that it reduced soft-tissue flare-ups significantly and prevented new areas of abnormal bone formation in patients with fibrodysplasia ossificans progressiva (FOP).
An international phase 3 clinical trial for a rare, inherited form of ALS showed that an investigational drug, known as tofersen, reduced molecular signs of disease, but at six months did not improve ...
Poor sleep is associated with a significantly increased risk of life-threatening flare-ups in people with chronic obstructive pulmonary disease, or COPD, according to a new study. The risk for these ... In an international phase 1--2 clinical trial of patients with immune thrombocytopenia, an oral investigational drug called rilzabrutinib was active and associated with only low-level toxic effects ...
A mutation in the gene that causes fibrodysplasia ossificans progressiva doesn't just cause extra bone growth but is tied to a problem in generating new muscle tissue after injury, ...
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