Nearly two-thirds of adult patients on crinecerfont achieved glucocorticoid doses within the physiologic range, compared to just 18% on placebo. The US Food and Drug Administration (FDA) has approved ...

Physiotherapy is the best way to keep children with DMD on their feet as long as possible, but my grandson is just a boy who wants to play. A New Genetic Test Clarified My Muscular Dystrophy Diagnosis ...

Test Your Knowledge About the Signs and Symptoms of FNAIT Test Your Knowledge About the Epidemiology of FNAIT Test Your Knowledge About the Diagnosis of FNAIT Rare Disease Advisor, a trusted source of ...

Fetal and neonatal alloimmune thrombocytopenia (FNAIT) is a rare blood condition that develops in the fetus or neonate during pregnancy or at birth in which maternal alloantibodies target paternally ...

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

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...

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 Knowledge About Panzyga for the Treatment of CIDP Test Your Knowledge About Life Expectancy for CIDP Test Your Knowledge About Clinical Features of CIDP ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

Get the latest news, resources, and perspectives on idiopathic pulmonary fibrosis (IPF), written specifically for HCPs who treat the disease.

The core care team for TK2d may include a neuromuscular neurologist, pulmonologist, nutritionist, physical therapist, and orthopedist. A support care team may include specialists such as a ...

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 ...