We expect to exit this year with three gene therapy programs in clinical trials - for Fabry,
Gaucher and cystinosis," commented CEO Geoff MacKay.
M2 PHARMA-November 28, 2018-Orphazyme announces publication of manuscript on preclinical Proof-of-Concept of arimoclomol in
Gaucher disease in EBioMedicine journal
M2 EQUITYBITES-November 28, 2018-Orphazyme announces publication of manuscript on preclinical Proof-of-Concept of arimoclomol in
Gaucher disease in EBioMedicine journal
Global Banking News-November 28, 2018-Orphazyme announces publication of manuscript on preclinical Proof-of-Concept of arimoclomol in
Gaucher disease in EBioMedicine journal
NORDIC BUSINESS REPORT-June 22, 2018-Orphazyme enrols first patient in phase II clinical trial for
Gaucher disease
Perinatal lethal
Gaucher disease (PLGD) is a rarer variant of type 2
Gaucher disease (GD).
There are around 7000 rare diseases but patients suffering from a particular segment of rare diseases called Lysosomal Storage Disorders (LSDs) like MPS (type I, II, III, IV & VI),
Gaucher, Pompe, Fabry, etc.
Introduction: The
Gaucher disease is a metabolic disorder due to deficiency or absence of acid [beta]-glucosidase enzyme.
Although
Gaucher disease generally presents with massive splenomegaly, which one of the predisposing causes of a wandering spleen, literature shows only one report of a wandering spleen in a child with
Gaucher disease.
(Bone impairment in patients with
Gaucher disease diagnosis).
Mesenteric lymphadenopathy is a rare manifestation of
Gaucher disease (GD) with only 26 cases reported worldwide and its outcome remains largely unknown.