Diet and nutrition play a key role in managing Duchenne muscular dystrophy (DMD). Dietary goals include preserving lean muscle mass, preventing weight gain and fat accumulation, maintaining bone ...

Monomeric C-reactive protein (mCRP) levels were significantly elevated in AAV and declined earlier than pentameric CRP during treatment.

An extension of the dosing interval of lanadelumab retains effectiveness in reducing attack frequency in patients with HAE.

Bisphosphonate scintigraphy has a high diagnostic accuracy in ATTR-CM caused by a Glu54Gln mutation in the TTR gene.

Researchers analyzed the applicability of the revised IWG-ELN criteria in the setting of ruxolitinib-treated patients with MF.

A new dual-feature framework was able to increase the diagnostic ability of myeloproliferative neoplasms, including MF, a study found.

Investigational gene therapy showed sustained clinical benefit and long-term safety in severe hemophilia B over 13 years of follow-up.

The incidence of TLS and associated mortality among hematological cancers, including acute lymphoblastic leukemia (ALL), is on the rise.

Keyana Sullivan, diagnosed with Pompe disease, uses a wheelchair and is dependent on a ventilator. Nonetheless, she is very goal-oriented. Keyana is a creative writer with a bachelor's degree in ...

Two metabolites, epiandrosterone sulfate and n-butyl oleate, demonstrated robust causal associations with idiopathic pulmonary fibrosis. Endogenous metabolic profiles may influence the pathogenesis of ...

Audiovestibular abnormalities such as sensorineural hearing loss are common in Gaucher disease types 1 and 3, according to new research.

Adding cyclophosphamide to glucocorticoids did not reduce relapse risk at 12 months in EGPA patients without poor prognosis factors.