Results 231 to 240 of about 415,200 (261)

Italian Patients Journey for Gene Therapy in Haemophilia A

open access: yesHaemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno   +19 more
wiley   +1 more source

Rare Variants in PFIC‐Related Genes Among Adults With Intrahepatic Cholestasis

open access: yesHepatology Research, EarlyView.
ABSTRACT Aim Biallelic pathogenic variants in progressive familial intrahepatic cholestasis (PFIC)‐related genes cause severe pediatric cholestasis. However, the clinical significance of heterozygous variants in adult intrahepatic cholestasis remains unclear.
Shunji Hirose   +9 more
wiley   +1 more source

Clinical Characteristics and Technical Outcomes of Microballoon Catheter-assisted Partial Splenic Embolization. [PDF]

open access: yesIn Vivo
Ishikawa T   +10 more
europepmc   +1 more source

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