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Latest News

Onasemnogene Abeparvovec Effective at Improving Motor Skills in SMA Type 1

Study Slated to Test New Rehabilitation Method in MS

Study Identifies Biomarkers of Emphysema in AATD and COPD

HAE Attack Severity Associated With Alterations in Throat Microbiota

USP13 May Play a Protective Role in Idiopathic Pulmonary Fibrosis

Routine Autoantibody Level Testing Not Recommended in MG

Three Weeks of Rehabilitation Improves Walking Ability in MS, Study Finds

Case Report: Patient With Hereditary Angioedema Dies After COVID-19 Infection

Sexual Dysfunction Is Significant, but Underrecognized, Among Women With SCD

Targeting Orai1: A Potential Therapeutic Strategy in PAH

Systematic Review Aims to Identify Clinical Characteristics of Ocular MG

Hip Pain Often Reported in SMA Type 2 Prior to Disease-Modifying Therapy

New Trial Sets Out to Evaluate Mitoxantrone Hydrochloride in NMOSD

High Pain Incidence Identified Among Patients With NMOSD

DMD May Be Associated With Language Development Abnormalities

Latest Features

The Danger of Having Pneumonia With Pompe Disease

As a person living with Pompe disease, I’ve had pneumonia at least 4 times in my life, and every time I landed in the hospital for weeks.
The Mental Toll of the COVID-19 Pandemic in Patients With Hereditary Angioedema

Research suggests mental health issues due to concerns about the COVID-19 pandemic can exacerbate symptoms of hereditary angioedema.
The Many Potential Ocular Manifestations in Patients With Sickle Cell Disease

Proliferative sickle retinopathy is the main cause of vision loss in patients with SCD, but the disease can affect virtually any part of the eye.