The global prevalence of PNH is as high as 15.9 individuals per 1 million people. The annual global incidence of PNH is around 5 to 6 individuals per 1 million people. 5,6 In the United States, ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

As of May 2025, 62 active clinical trials are ongoing for Fabry disease, including 25 interventional and 37 observational studies, of which 8 are patient registries. Among the trials with known ...

Biochemical response rates were sustained through week 182 in patients with PBC treated with elafibranor, and the proportion of patients with normal alkaline phosphatase levels remained consistent.

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

Sharon Hesterlee, PhD, who previously served as chief research officer at the MDA, has been named interim president and CEO. Donald S. Wood, PhD, has retired from the Muscular Dystrophy Association ...

The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with Duchenne muscular dystrophy (DMD), according to a new study ...

Belinda Moser of ExamOne takes a blood sample from Marie Bedirlian, whose 14-year-old son, Brayden, has Duchenne. Bedirlian is a DMD carrier. (Photo by Larry Luxner) Pradeep Mammen, MD, an expert on ...

Efficacy analyses for the first 2 cohorts of the phase 1/2 studies will compare participant outcomes with those of a propensity score-matched external control group. AMT-130, an investigational gene ...

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...

Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...

BLANKENBERGE, Belgium—Herwig Lange, MD, has been researching Huntington disease (HD) since 1969. And he’s convinced that avoiding stress, eating healthy, and getting lots of exercise can delay the ...