Rare Disease Advisor

Elafibranor Has Similar Safety Profile to Placebo in Patients With PBC

Patients with PBC treated with elafibranor had similar rates of adverse events to those treated with placebo, based on a meta ...
Rare Disease Advisor

Statins Linked to Slower Progression and Lower Mortality in Non-Cirrhotic PBC

Statins were associated with fewer cases of cirrhosis, liver decompensation, and death in patients with PBC who did not yet ...
Rare Disease Advisor

What Does Independence Look Like for a Person With PWS?

A parent wants medical professionals who treat children with PWS to understand the challenges of raising these kids to be ...
Rare Disease Advisor

Innovative Oxford-Harrington Partnership Amplifies Rare Disease Research Funding

The partnership has already brought in more than $200 million in philanthropic donations to help fund specific rare disease ...
Rare Disease Advisor

Study Underscores Challenges in Maintaining Nintedanib Dosage in PPF

Modifications to nintedanib treatment are common and usually due to tolerability issues in patients with PPF, research shows.
Rare Disease Advisor

Automated CT Scoring Can Predict Progression of Interstitial Lung Abnormalities

Usual interstitial pneumonia–like computed tomography features are associated with fibrosis progression in interstitial lung ...
Rare Disease Advisor

Echocardiographic Stability Observed With Tafamidis in ATTR-CM

In a recent study, tafamidis-treated patients with wild-type ATTR-CM demonstrated numerically stable serial echocardiographic ...
Rare Disease Advisor

LVH–QRS Voltage Discordance Marks Worse Myocardial Function in ATTR-CM

A mismatch between LVH and QRS voltage identifies a wild-type ATTR-CM phenotype linked with higher heart failure risk, ...
Rare Disease Advisor

ATTR-CM Frequently Coexists With Plasma Cell Dyscrasias, Case Series Shows

Clinicians should not assume that suspected cardiac amyloidosis in patients with plasma cell dyscrasias is always AL-CM, ...
Rare Disease Advisor

Concurrent Sjögren Disease and PBC Complicate Arthritis Diagnosis

Sicca symptom screening and anti-Ro/SSA testing may help distinguish Sjögren disease with PBC from RA in patients with ...
Rare Disease Advisor

High Mortality Rates Reported in Patients With Autoimmune ILD and PPF

Patients with autoimmune interstitial lung disease (ILD) and PPF have substantial mortality related to ILD, a study shows.
Rare Disease Advisor

Air Pollution May Be Associated With Respiratory Hospitalization in IPF

Short-term increases in PM2.5 are associated with a higher risk of respiratory hospitalization among patients with IPF, ...