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2 articles from the last 30 days matching "chaple disease"

Clinical trialRSS3 days ago

5-year survival seen for over 40% of ALS patients on masitinib in trial

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.
💬 Ask your doctoramyotrophic lateral sclerosisRead →
Clinical trialCLINICALTRIALSApr 14

Trial Now Recruiting: A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease (NCT07142343)

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.
→ You can act on thisCHAPLE diseaseRead →

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