Patients witll either receive 55 mg of nexiguran ziclumeran or a placebo via a single intravenous infusion. A new phase 3 clinical trial testing the safety and efficacy of nexiguran ziclumeran ...

Physical limitations and pain in achondroplasia often impair patients’ ability to perform activities of daily living. Adult patients with disproportionate short-stature skeletal dysplasia (SD) such as ...

These findings reveal that a new NMOSD-specific tool to determine disability could be beneficial for this population. Patients with neuromyelitis optica spectrum disorder (NMOSD) could benefit from a ...

A phase 2a trial reports a height growth rate increase of up to +4.6 cm/year in pediatric patients with achondroplasia receiving low-dose umedaptanib pegol. Interim results from a phase 2a clinical ...

The mean annual total costs were highest with very severe disease at €19,570 to €33,495 per person across all 3 countries. Myasthenia gravis (MG), especially in its severe form, leads to a high level ...

Results of the receiver operating characteristic curve analysis revealed that CD63 expression level could distinguish ITP patients from those with other causes of thrombocytopenia. Two immunological ...

One case of intraparenchymal extranodal marginal zone lymphoma (EMZL) showed plasmacytic differentiation and had pathogenic mutations in the SPEN and ARID1A genes. A team of researchers from Spain ...

While tracheostomy placement is common in skeletal dysplasia, those patients with achondroplasia are less likely to need a tracheostomy. A new retrospective review of patients with skeletal dysplasia ...

Patients with ATTR-PN performed worse in executive functions as well as verbal and visual memory, visuospatial domains, and language tests. The thickness of the cortex in the inferior temporal gyrus, ...

Timely surgical interventions are key to resolving neurological deficits in cases of disc herniations associated with achondroplasia. A patient with an established diagnosis of achondroplasia received ...

Patients with rare diseases, like hereditary angioedema (HAE), who live in rural areas often face obstacles to care. Healthcare inequalities have always existed; the only question of importance is the ...

Which of the statements below about the risk factors for sickle cell disease (SCD) are correct? Select one of the options for each question, then submit your answers to see how well you scored and how ...