Foramen magnum development may be largely determined in utero, potentially explaining why postnatal vosoritide was unable to prevent stenosis. Two infants with achondroplasia who initiated vosoritide ...

Vesna Aleksovska knows of only 15 other people with Gaucher disease in her country of 1.8 million people. Rare Disease Advisor, a trusted source of medical news and feature content for healthcare ...

Selinexor in combination with ruxolitinib achieved a statistically significant improvement in spleen volume reduction for patients with MF, according to a press release. Rare Disease Advisor, 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 ...

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 ...

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 ...

In patients with neuromyelitis optica spectrum disorder (NMOSD), treatment with the IL-6 receptor monoclonal antibody satralizumab was associated with a markedly lower relapse rate compared to ...

Imagine getting the chance to eat your favorite meal, but on your first bite, you struggle to chew, and your eyes suddenly water as the food gets stuck in your throat as you swallow. Struggles with ...

Further research is needed to establish if there is a causative link between avacopan and liver injury. A patient with microscopic polyangiitis (MPA)—a form of antineutrophil cytoplasmic antibody ...

Kerrie Walters, who has MS, and her service dog, Wooldridge, at home in Grants Pass, Oregon (Credit: Jennifer Butler Photography) Kerrie Walters first heard of PoNS in a chance encounter with TV ...

Consolidation or maintenance therapy significantly improved progression-free survival and overall survival. Zanubrutinib, lenalidomide, temozolomide, and rituximab (RLZT) combined with or without high ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...