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 ...

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 ...

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 ...

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 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 ...

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 ...

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 ...

Several factors influence the prognosis of gastrointestinal stromal tumors (GISTs), but the primary prognostic factors include the tumor staging and the tumor resectability. The staging for GISTs ...

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 ...

Test Your Knowledge About the Differential Diagnosis of LAL-D Test Your Knowledge About the Guidelines for LAL-D Test Your Knowledge of Enzyme Replacement Therapy for ...