Get the latest news, resources, and perspectives on Duchenne muscular dystrophy (DMD), written specifically for HCPs who treat the disease.

Follow the latest news, insights and perspectives on the treatment of Chronic Inflammatory Demyelinating Polyneuropathy at Rare Disease Advisor.

The change in extracellular volume is the only predictor of adverse outcome in patients with ATTR-CM treated with tafamidis, a study found.

Get the latest news, resources, and perspectives on spinal muscular atrophy (SMA), written specifically for HCPs who treat the disease.

Are you a health care provider looking for information on sickle cell disease life expectancy? Read more about it here.

Alithea is a mother of 3 young adults and works for a nonprofit advocating for healthier lives. In 2020, she was diagnosed with cold agglutinin disease (CAD), a rare form of hemolytic anemia. She ...

Results of the ApproaCH trial showed that navepegritide improved AGV over placebo in pediatric patients with achondroplasia.

Angela Schaff, a single mother of 3 boys, has been a stay-at-home mom since 2005, when she started to have medical issues. Those issues culminated when she was diagnosed with primary biliary ...

Several investigational therapies are being evaluated for the treatment of Prader-Willi syndrome, a rare disease marked by persistent hyperphagia.

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare, acquired neurological condition affecting the peripheral nervous system in which inflammatory immune mechanisms target and damage ...

Potential new biomarkers of LGS were discovered using unsupervised time-frequency analysis of EEG recordings collected during sleep, a study found.

Landiolol has been approved for treating supraventricular tachycardia—a frequent and serious complication of pulmonary arterial hypertension (PAH).