Test Your Knowledge About the Signs and Symptoms of HDFN Test Your Knowledge About the Guidelines for HDFN Test Your Knowledge About Therapies for HDFN Test Your ...

Test Your Knowledge About Eteplirsen for the Treatment of DMD Test Your Knowledge About the Treatment of DMD Test Your Knowledge About the Epidemiology of DMD Test Your Knowledge About the ...

There were significant associations between echocardiographic parameters, such as increased left ventricular mass and greater interventricular septal thickness at end-diastole, and positive 99mTc-PYP ...

The registry’s primary outcome measure is the prevalence of the systemic form of mastocytosis in the studied population. A new single-center longitudinal registry of patients with cutaneous and ...

At the time of diagnosis, patients with different types of [cardiac amyloidosis] had distinct echocardiographic presentation and survival differed in the first 3 years. Differences in ...

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, ...

Overall, the life expectancy of patients with SCD is lower than that of persons without SCD. 1 However, life expectancy varies depending on the type of disease, treatments administered, and ...

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 ...

Medullary thyroid cancer (MTC) is a type of thyroid cancer that affects thyroid gland cells called the parafollicular C cells. 1 These cells are responsible for making the hormone calcitonin, which ...

Patients with immune thrombocytopenia (ITP) who are positive for hepatitis B core antibodies (anti-HBc) exhibit a more severe disease course.

Jerry Lewis, one of the most famous comedians of the 20th century, was a tireless advocate for people with muscular dystrophy. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

The term muscular dystrophy encompasses a group of genetic diseases characterized by progressive muscle weakness and wasting. There are different types of muscular dystrophy depending on genetic ...