Intravenous immunoglobulin therapy, a key treatment used in managing FNAIT, has been linked to the development of thrombocytopenia.

Therapies for Gaucher disease generally fall into 2 main categories: enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Neither ERT nor SRT effectively treats the neurological ...

A new study conducted in India has revealed that 40% of antenatal patients experienced severe fetomaternal hemorrhage, which can increase the risk of HDFN.

Large language models may offer complementary value to human expert analysis in the diagnosis of achondroplasia and other skeletal dysplasias.

Forskolin may help increase the regeneration of the muscle tissue and reduce fibrosis in Duchenne muscular dystrophy (DMD), a study found.

The FDA approved Y-90 resin microspheres for targeted radiation treatment of unresectable hepatocellular carcinoma (HCC).

Compound 23, a dual-target FGFR2/3 inhibitor, delivered superior growth improvements over infigratinib in achondroplasia mouse models.

A clinical scoring system has been validated for predicting 30-day mortality in acute exacerbations of idiopathic pulmonary fibrosis (IPF).

Long-term prophylaxis with lanadelumab has been linked to a marked reduction in attack rates in HAE, with the decline persisting in a real-world setting.

Frailty reduced the likelihood of CLL/SLL first-line treatment initiation during the CIT era, but the gap narrowed with novel therapies.

Biomarkers are increasingly recognized for their use in diagnosing and tracking disability progression in NMOSD.

TTR amyloid deposition was present in the vessel walls of different organs throughout the body in ATTR-CM and Alzheimer’s disease, a study found.