#Rituximab did not significantly improve remission rates compared with conventional therapy in patients with eosinophilic granulomatosis with polyangiitis (#EGPA). Annals of Int Med Read More: bit.ly/46ROT5k #RareDisease

Currently available treatments slow the progression of kidney and heart disease associated with #FabryDisease, according to a systematic review of the literature. JCM MDPI Read More: bit.ly/3J7Sum5 #RareDisease

Surgery for patients with #Achondroplasia should involve careful preoperative preparation and the use of specialized positioning equipment. AfPP - SaferSurgeryUK Read More: bit.ly/45rqRf6 #RareDisease

A composite measure of lung function, imaging, and blood biomarkers holds promise to improve prediction of mortality in #IdiopathicPulmonaryFibrosis (IPF). BMC Read More: bit.ly/4ltkcHo #RareDisease

Elevated levels of interleukin-10 (IL-10) in cerebrospinal fluid (CSF) may serve as a diagnostic biomarker in primary central nervous system lymphoma (#PCNSL). Cureus Read More: bit.ly/45hOvvW #RareDisease

Attenuated lysosomal storage disorders often present in adulthood with subtle cardiovascular, neurological, and respiratory findings that delay recognition. Genes MDPI Read More: bit.ly/4fyLHxT #RareDisease #GaucherDisease

Encouraging preclinical data were announced for the investigational medication EVT0185 in #HepatocellularCarcinoma (HCC). Read More: bit.ly/3UnbcIK #RareDisease

"Having #ColdAgglutininDisease (CAD), a form of autoimmune hemolytic anemia that is cold reactive, makes managing pain difficult." - Alithea Athans, CAD Contributor Read More: bit.ly/45vbwKv #RareDisease

The combination therapy of pozelimab and cemdisiran resulted in adequate hemolysis control in patients with #ParoxysmalNocturnalHemoglobinuria (PNH), according to a study published in eJHaem Journal. Read More: bit.ly/3Hntzuc #RareDisease

Nipocalimab shows promise for improving pregnancy safety and neonatal outcomes in both #HDFN and #FNAIT by blocking the neonatal Fc receptor. ISPD Read More: bit.ly/46QAztV #RareDisease

"I was supposed to be dead by now, or so statistics said." - Ed Tobias, MS Contributor Read More: bit.ly/4oyEfqB #RareDisease #MultipleSclerosis

Addition of PD-1 inhibitors to lenvatinib plus TACE significantly improved clinical response in intermediate or advanced #HepatocellularCarcinoma (HCC). Frontiers - Immunology Read More: bit.ly/41JRURJ #RareDisease

The Toronto Criteria for treatment response at 6 months helps predict complete biochemical response (CBR) and overall prognosis in patients with #PrimaryBiliaryCholangitis (PBC), a study found. BMC Read More: bit.ly/4mODnMZ #RareDisease

Non-coding RNAs play a role in the pathogenesis of #PrimaryBiliaryCholangitis (PBC) and may represent diagnostic and therapeutic targets, a study found. Frontiers - Medicine Read More: bit.ly/4mFxUHU #RareDisease

Delayed CAR T-cell infusion after apheresis is an independent poor prognostic factor in diffuse large B-cell lymphoma (#DLBCL). BSH - Haematology Read More: bit.ly/3UuAqVI #RareDisease

Differentiated keratinocytes may release molecules that degrade or block harmful neutrophil extracellular trap (NET) structures in #GeneralizedPustularPsoriasis (GPP). Society for Investigative Dermatology Read More: bit.ly/3V6W9D6 #RareDisease

#GeneralizedPustularPsoriasis (GPP) has a profound impact on patients’ quality of life (QoL) and psychological well-being, according to findings of a recent study published in Dermatology and Therapy. Dermatology and Therapy Read More: bit.ly/3V0dWfh #RareDisease

Researchers evaluated treatment response, survival outcomes, and the safety of various midostaurin dosing regimens in a cohort of patients with advanced #SystemicMastocytosis (SM). British Journal of Haematology Read More: bit.ly/4oCFXqX #RareDisease

Early diagnosis in #SystemicMastocytosis (SM) with acute myeloid leukemia can lead to initiation of appropriate therapies that increases the odds of a more favorable outcome. Pathology Journal Read More: bit.ly/4mN8nNi #RareDisease

#IgG4-related disease was marked by reduced galactosylation and elevated sialylation, confirmed across independent patient cohorts. Journal of Proteome Research Read More: bit.ly/46YfNIN #RareDisease