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

3 articles from the last 30 days matching "blood test"

ResearchRSSMay 7

Prebiotics balance gut bacteria and show results in Parkinson’s blood

Researchers found that a special type of food supplement called a prebiotic can help restore healthy bacteria in the guts of people with Parkinson's disease. Even better, they discovered a simple blood test that can measure tiny particles to show whether the treatment is working. This blood test tracks signals that travel between the gut and the brain.

WHY IT MATTERSFor Parkinson's patients, this offers a non-invasive way to monitor whether gut-targeted treatments are actually working, potentially opening a new avenue for managing symptoms through gut health.
💬 Ask your doctorParkinson's disease
ResearchPUBMEDMay 1

The Placebo Effect in Rare Disease Clinical Trials: Measurement, Impact, and Statistical Approaches for Patient-as-Own-Control Designs.

Researchers studied whether the placebo effect (feeling better just because you expect to) is a real problem in rare disease trials where patients serve as their own comparison group. They found that for objective measurements like blood tests and imaging scans—which are used in most approved rare disease treatments—the placebo effect is very small and usually not a major concern.

WHY IT MATTERSIf you're considering enrolling in a rare disease trial using a patient-as-own-control design, this research shows that the results are likely measuring real treatment effects rather than just placebo effects, making the trial data more trustworthy for evaluating whether a treatment actually works.
ℹ Good to know
ResearchRSSApr 29

Corvista, Mayo Clinic team up to study non-invasive PH diagnostic test

Corvista Health and Mayo Clinic are working together to test a new blood test that could help doctors diagnose pulmonary hypertension (a serious lung disease where blood pressure in the lungs gets too high) without invasive procedures. The test aims to make it easier to identify who has the disease, figure out how severe it is, and decide who needs to see a specialist. This collaboration is an important step forward in developing better diagnostic tools for this condition.

WHY IT MATTERSIf successful, this non-invasive test could help patients with suspected pulmonary hypertension get diagnosed faster and more accurately, potentially avoiding unnecessary invasive procedures like heart catheterization while ensuring those who need specialist care are properly referred.
👁 Watch this spacepulmonary hypertension

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