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

6 articles from the last 30 days matching "pah"

ResearchRSS3 days ago

RUNX1 gene may serve as biomarker for right heart failure in PAH

Scientists found that a gene called RUNX1 might help doctors predict when patients with pulmonary arterial hypertension (PAH) — a serious lung disease that makes it hard to pump blood — could develop heart problems. When the right side of the heart weakens in PAH patients, it becomes very dangerous. This discovery could help doctors catch and treat these heart problems earlier.

WHY IT MATTERSIf RUNX1 becomes a validated biomarker, PAH patients could receive earlier interventions to prevent right heart failure, potentially improving survival and quality of life before irreversible damage occurs.
Good to knowpulmonary arterial hypertensionright heart failureRead →
NewsRSS3 days ago

Test Your Knowledge About Riociguat for the Treatment of PAH

This article is a quiz that tests what you know about riociguat, a medication used to treat pulmonary arterial hypertension (PAH). PAH is a rare condition where blood vessels in the lungs become narrowed, making it harder for the heart to pump blood through them. The quiz helps patients and caregivers learn more about how this treatment works.

WHY IT MATTERSUnderstanding how riociguat works and its role in PAH treatment can help patients make informed decisions about their care and have more meaningful conversations with their doctors about their treatment options.
Good to knowpulmonary arterial hypertensionRead →
Clinical trialRSS3 days ago

First patient enrolled in Phase 3 trial of PAH treatment IKT-001

A new clinical trial has started testing a drug called IKT-001 in people with pulmonary arterial hypertension (PAH), a rare condition where blood vessels in the lungs become narrowed and make it hard for the heart to pump blood. This is a large Phase 3 trial, which is a late-stage test that happens before a drug might be approved. Nearly 500 adults will participate to see if this drug works better than current treatments.

WHY IT MATTERSIf you have PAH and are already on stable treatment, you may be eligible to join the IMPROVE-PAH trial and potentially access IKT-001 before it becomes widely available.
You can act on thispulmonary arterial hypertensionRead →
NewsRSS3 days ago

Orenitram (oral treprostinil) for pulmonary arterial hypertension

Orenitram is a pill form of a medicine called treprostinil that helps people with pulmonary arterial hypertension (a serious lung condition where blood vessels in the lungs become too narrow). You take it by mouth 2-3 times a day with food. This medicine helps patients exercise better and slows down how fast their disease gets worse.

WHY IT MATTERSThis oral option gives PAH patients an alternative to injected or inhaled treprostinil, making treatment more convenient for daily management.
💬 Ask your doctorpulmonary arterial hypertensionRead →
ResearchRSS3 days ago

High pulmonary resistance indicates worse outcomes in new PH study

A study from Japan found that people with pulmonary hypertension (a condition where blood vessels in the lungs become stiff and narrow) who have higher pulmonary vascular resistance—a measure of how hard the heart has to work to pump blood through the lungs—tend to have worse health outcomes. The study also looked at whether a type of diabetes medication called SGLT2 inhibitors might help these patients.

WHY IT MATTERSIf you have pulmonary hypertension related to heart disease, knowing that high pulmonary vascular resistance predicts worse outcomes could help your doctor decide whether to start more aggressive treatment earlier or monitor you more closely.
💬 Ask your doctorpulmonary hypertension due to left heart diseaseRead →
ResearchRSS3 days ago

Smartphone data may help detect rare lung disease earlier, study suggests

Researchers found that data from smartphones and wearable devices, collected over several years through a health app, can help doctors identify people with a rare lung disease called idiopathic pulmonary arterial hypertension (IPAH) earlier than before. The study used information like heart rate and activity levels from the My Heart Counts app to spot differences between people with IPAH and healthy people. This discovery could lead to faster diagnosis of this serious condition.

WHY IT MATTERSEarlier detection of IPAH could allow patients to start treatment sooner, potentially slowing disease progression and improving outcomes for this condition that currently has no cure.
Good to knowidiopathic pulmonary arterial hypertensionRead →

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