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 ...

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 ...

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 ...

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 ...

From landmark gene therapy approvals to illuminating discoveries into the pathogenesis of rare diseases, 2023 saw substantive advances in rare disease diagnosis and treatment. The following list of ...

A diagnosis of ATTR-CM in a more recent era was associated with older age, White race, wild‐type disease, and tafamidis treatment. The survival of patients diagnosed with transthyretin-mediated ...

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 ...

Besides the year I received my diagnosis, 2024 has been the biggest year that primary biliary cholangitis (PBC) has affected me thus far.

There are a number of guideline recommendations available by the American Academy of Neurology (AAN) for the evaluation, diagnosis, management, treatment, and care of patients affected by muscular ...

Muscular dystrophy is the name given to a group of genetic diseases characterized by progressive muscle weakness and wasting. There are several types of muscular dystrophy, all with a different ...

A pattern recognition approach takes into account factors like disease progression rate, whether the disease symptoms are symmetrical or asymmetrical, whether they are predominantly distal or proximal ...