Given the results, the investigation of intranasal carbetocin in PWS has been halted. Intranasal carbetocin did not demonstrate clinical benefit for hyperphagia in patients with Prader-Willi syndrome ...

The study highlights the benefits a robust genetic testing program can have, which ultimately has the potential to improve outcomes ATTRv is caused by more than 130 known mutations in the TTR gene and ...

The consequences of cystic fibrosis (CF) are extensive and affect almost every organ system. The clinical picture is broad and may present with acute or chronic manifestations. Gastrointestinal and ...

Momelotinib treatment leads to clear clinical benefits in patients with myelofibrosis (MF), according to results from a real-world study. Rare Disease Advisor, a trusted source of medical news and ...

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 ...

In diagnosed cases of cutaneous mastocytosis, investigations to rule out systemic mastocytosis may be appropriate. A young child with a small chest lump and anemia was found to have cutaneous ...

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 ...

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 ...

My recent liver biopsy was a case study of Murphy’s Law in action. And as I share the details of my saga, I’m mindful that my experience is the exception, not the rule. I do not want to discourage ...

While being diagnosed with immune thrombocytopenia (ITP) has everything to do with our physical body, it can also have a severe effect on our mental and emotional health. It’s almost as if you go ...

Positive topline data from a phase 1 clinical study (NCT04814498) of cudetaxestat (BLD-0409) have been presented by Blade Therapeutics, a South San Francisco biopharmaceutical company. The study ...

During the last 4 years of my immune thrombocytopenia (ITP) journey, I have tried natural remedies to normalize my platelet levels. Most people don’t want to take medication unless they absolutely ...