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Latest News

Antibody Status Impacts Disease Severity in MG

Elevated Plasma C5a Levels Correlate With Pain in NMOSD

AAVs Appear to Be Effective Vectors for Gene Therapy in LCFAOD

Older Patients With Hemophilia Show Mild Disease But Poor Quality of Life

New Gene Therapy Approach Holds Potential Therapeutic Value in LAL-D

Poor Response to Nusinersen May Predict Benefit From Onasemnogene Abeparvovec

Teriflunomide Use Increasing in Patients With MS, Particularly Among Some Subgroups

Rodatristat Ethyl Plus Ambrisentan Reduces Occlusions in PAH Model

Cognitive Performance Predicts Self-Management in Patients With MS

High KL-6 Level Associated With Disease Progression in IPF

Researchers Recommend Adding 3D FLAIR Sequences to MRI Protocol for MS

Study Highlights Need to Manage Sinonasal Symptoms in CF

Cognitive Impairment Correlates With Decreased Thalamic Volume in MS

Rapid Whole Genome Sequencing Facilitates Early Treatment of LAL-D

Newly Identified Metabolites Aid in Early Detection of MTC

Latest Features

Should I Have Another Catheterization for My Pulmonary Arterial Hypertension?

I live with pulmonary arterial hypertension (PAH), a progressive rare disease that is treated by addressing the symptoms only and not the disease.
The Underrecognized Burden of Sleep‐Disordered Breathing in Cystic Fibrosis

The underdiagnosis and consequent undertreatment of sleep-disordered breathing in the CF population can compromise patients’ overall outcomes.
Aytu BioPharma’s Healight Device Could Help Treat Rare Pulmonary Diseases

Aytu BioPharma has developed an endotracheal respiratory catheter which its CEO says has “tremendous potential” in IPF, PAH, and other respiratory diseases.