Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

2 articles from the last 7 days matching "sma"

Clinical trialCLINICALTRIALSYesterday

Trial Now Recruiting: A Study to Evaluate the Efficacy and Safety of Sutetinib Maleate Capsule in Locally Advanced or Metastatic NSCLC (NCT06010329)

Researchers are testing a new cancer drug called sutetinib maleate in people with advanced lung cancer that has specific uncommon genetic mutations. This is a Phase 2 trial, meaning the drug has already been tested in a small group and now researchers want to see if it works better and remains safe in a larger group of 66 patients. The study is currently accepting new participants.

WHY IT MATTERSIf you have metastatic or locally advanced non-small cell lung cancer with uncommon EGFR mutations, this trial offers access to a potentially new treatment option that may not yet be available outside of clinical research.
You can act on thisnon-small cell lung cancerlocally advanced non-small cell lung cancermetastatic non-small cell lung cancerRead →
Clinical trialCLINICALTRIALS5 days ago

Trial Now Recruiting: Williams Syndrome (WS) and Supravalvar Aortic Stenosis (SVAS) DNA and Tissue Bank (NCT02706639)

Williams syndrome is a rare genetic condition that affects about 1 in every 8,000 to 12,000 people. It happens when a person is missing a small section of DNA on chromosome 7, which includes the gene that makes elastin (a protein that helps blood vessels stretch). This causes distinctive facial features and heart and blood vessel problems. Researchers are now recruiting 1,099 people with Williams syndrome or a related heart condition called supravalvular aortic stenosis to donate DNA and tissue samples to help scientists better understand these conditions.

WHY IT MATTERSThis actively recruiting biobank offers Williams syndrome and SVAS patients a direct way to contribute DNA and tissue samples to NHLBI-sponsored research that could accelerate understanding of the genetic causes and lead to new treatments.
You can act on thisWilliams SyndromeSupravalvular Aortic StenosisRead →

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases