BioMarin Pharmaceutical’s strong rare disease drug portfolio and Amicus acquisition drive a ‘Buy’ rating. Click here to read my latest analysis of BMRN stock.
For a child with a rare disease, a doctor’s continuing education can end a years-long diagnostic odyssey and unlock access to ...
ABSTRACT: Introduction: Dysphagia is an alarming symptom that requires upper gastrointestinal endoscopy to determine its etiology. The aim of this study was to assess the diagnostic and therapeutic ...
This study offers important insight into the pathogenic basis of intragenic frameshift deletions in the carboxy-terminal domain of MECP2, which account for some Rett syndrome cases, yet similar ...
JCR Pharmaceuticals and Italfarmaco announced an exclusive licensing agreement for the development and commercialization of givinostat in Japan.
Elfabrio (R) (pegunigalsidase alfa for injection) is an enzyme replacement therapy indicated for the longterm treatment of adult patients with a confirmed diagnosis of Fabry disease (deficiency of ...
Several measurement techniques used in the life sciences gather data for many more variables per sample than the typical number of samples assayed. For instance, DNA microarrays and mass spectrometers ...
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