Clinical trialRSS3 days ago
A new drug called masitinib helped ALS patients live longer than expected. In a clinical trial, over 40% of patients treated with masitinib survived for at least 5 years from when their disease started. This is much better than the typical outcome, where fewer than 25% of ALS patients usually live that long.
WHY IT MATTERSIf masitinib gains approval, ALS patients may have access to a treatment that could significantly extend their survival time compared to current standard care.
Clinical trialRSS3 days ago
AB Science is preparing to test an experimental drug called masitinib for ALS (a disease that affects nerve cells that control muscles). To protect their investment in this large clinical trial, the company bought special insurance that would reimburse them if the trial doesn't go as planned or costs more than expected.
WHY IT MATTERSThis insurance arrangement signals that AB Science is confident enough in masitinib to move forward with Phase 3 testing in ALS patients, which is a critical step toward potentially bringing a new treatment option to people living with this progressive disease.
Clinical trialCLINICALTRIALSApr 14
Researchers are testing a new medicine called pozelimab in young children ages 1 to 5 who have CHAPLE disease, a rare inherited condition that affects the stomach, intestines, heart, and blood vessels. This study is checking whether the medicine is safe and well-tolerated in this young age group. CHAPLE disease can cause serious symptoms, so finding safe treatments for young children is important.
WHY IT MATTERSThis is the first safety trial of pozelimab in very young children (ages 1-5) with CHAPLE disease, offering families of affected toddlers a potential treatment option when few alternatives exist for this life-threatening condition.