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 ...

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 ...

Diagnosis of neurofibromatosis type 1 (NF1) requires a combination of clinical assessment, genetic testing, and imaging studies. 1 NF1 is a rare genetic disorder characterized by the development of ...

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 ...

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, ...

The diagnosis and management of CIDP are complex and best addressed by an interprofessional health care team that includes neurologists, neuromuscular specialists, physical medicine and rehabilitation ...

ATTR-CM is a genetic condition characterized by the destabilization of transthyretin and subsequent misfolding and aggregation into amyloid fibrils that results in abnormal deposition of amyloid in ...

As a patient with primary biliary cholangitis (PBC), I would never participate in a clinical trial for a new medication that would treat my PBC.

The ratio of posterior wall thickness to the minimum voltage of QRS complexes in limb leads and several parameters derived from electrocardiography could independently predict ATTR-CM. The ratio of ...

Between 21.4% and 57.8% of patients from a real-world ATTR-CM cohort would have been eligible to take part in clinical trials testing disease-modifying therapies. Inclusion and exclusion criteria used ...

Given that SMA is a genetic disorder, the incidence of the disease is more accurately related to the birth prevalence or number of children born with SMA during a given period of time. 1 Many studies ...