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

5 articles from the last 90 days matching "survival"

ResearchRSSYesterday

Keeping weight steady after tube feeding may help ALS survival

A study of 121 people with ALS found that those who kept their weight more stable after starting tube feeding lived longer. This was true even if they weren't eating a lot of calories when they first started the tube feeding. The finding suggests that preventing weight loss after tube feeding may help people with ALS live longer.

WHY IT MATTERSIf you or a loved one with ALS is considering or has started tube feeding, this research suggests that working with your care team to maintain steady weight—not just calorie intake—could potentially extend survival.
💬 Ask your doctorAmyotrophic Lateral SclerosisRead →
Clinical trialRSS2 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 →
ResearchPUBMEDMar 26

Long-term survival outcomes of female genital tract rhabdomyosarcoma in children, adolescents and young adults at a national rare disease diagnosis and treatment center in China.

Researchers in China studied 26 children and young adults (average age 8 years old) who had a rare cancer called rhabdomyosarcoma that started in the female reproductive organs. They tracked these patients for an average of nearly 5 years to see how well different treatments worked. This study helps doctors understand the best ways to treat this uncommon type of cancer in girls and young women.

WHY IT MATTERSThis is the first large study from China showing long-term survival rates for girls with genital rhabdomyosarcoma, which can help doctors worldwide improve treatment plans and give families more accurate information about what to expect.
💬 Ask your doctorRhabdomyosarcomaFemale genital tract rhabdomyosarcomaGenitourinary rhabdomyosarcomaRead →
ResearchPUBMEDMar 26

Evaluation of mixed response in tumor size and survival in patients with rare cancers treated with dual checkpoint inhibitor therapy (DART SWOG S1609).

Researchers studied how cancer tumors respond differently to a two-drug immunotherapy treatment (ipilimumab plus nivolumab) in patients with rare cancers. Some patients had some tumors shrink while others grew at the same time—called 'mixed response.' This study looked at 438 patients to understand how this mixed response affects how long patients survive and whether the cancer comes back.

WHY IT MATTERSUnderstanding mixed response patterns helps doctors better predict which rare cancer patients will benefit most from dual checkpoint inhibitor therapy and may improve how treatment success is measured beyond just tumor size.
💬 Ask your doctorrare cancersmelanomanon-small cell lung cancerRead →
ResearchCLINICALTRIALSMar 26

New Clinical Trial: Optimal Methods of Disease Progression and Survival Analysis in Children and Adults Patients With Adrenocortical Cancer (ACC) (NCT04358107)

Researchers are studying 1,000 children and adults with adrenocortical carcinoma (ACC), a rare cancer of the adrenal glands. They want to understand why some people survive longer than others and whether certain treatments work better. The average person with ACC lives about 14.5 months after diagnosis, but survival times vary widely.

WHY IT MATTERSThis study is actively collecting data that could help doctors better predict outcomes and choose more effective treatments for ACC patients, though it is not currently recruiting new participants.
💬 Ask your doctorAdrenocortical CarcinomaRead →

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