Results 311 to 320 of about 960,906 (340)

Narsoplimab Results in Excellent Survival in Adults and Children With Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy (TA‐TMA)

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Inappropriate complement activation is a key driver of hematopoietic cell transplant‐associated thrombotic microangiopathy (TA‐TMA). Treatment with narsoplimab, an inhibitor of MASP‐2, the effector enzyme of the lectin pathway, resulted in a response rate of 61% in a Phase 2 clinical trial in adults with TA‐TMA.
Michelle L. Schoettler   +6 more
wiley   +1 more source

Identification and Characterization of a Novel Biallelic SLC12A2 Variant Associated With Kilquist Syndrome (OMIM #619080)

open access: yesAmerican Journal of Medical Genetics Part A, EarlyView.
ABSTRACT This study presents the case of a child with multiple congenital anomalies, severe hypotonia, and profound bilateral sensorineural hearing loss. Functional bioenergetic assessments showed no significant mitochondrial respiratory defects, and riboflavin (Rf) status evaluation excluded a deficiency in Rf transporters as a cause of hearing loss ...
Piero Leone   +13 more
wiley   +1 more source

Recurrent immunotactoid glomerulopathy in a kidney transplant recipient: Case report. [PDF]

open access: yesMedicine (Baltimore)
Yu S   +6 more
europepmc   +1 more source

Psoriasis Does Not Seem to Impair Glomerular and Tubular Function - The Comprehensive Study on Serum and Urine. [PDF]

open access: yesPsoriasis (Auckl)
Nowowiejska J   +8 more
europepmc   +1 more source

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