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Trend Chart on Orphans – February 23rd, 2019
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Trend Chart on Orphans – February 23rd, 2019

FEATURE STORY 

● Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

GENE THERAPY

● Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

 

● Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9

RARE DISEASES

● Characterization and treatment of congenital thrombotic thrombocytopenic purpura

● Targeting angiogenesis in Duchenne muscular dystrophy

­CLINICAL TRIALS – DATA

●  Catabasis Pharmaceuticals: new data supporting edasalonexent as a potential treatment for Duchenne Muscular Dystrophy

INDUSTRIAL LANDSCAPE &AGREEMENTS

● AOP Orphan announces EU marketing authorization for ropeginterferon alfa-2b for rare blood cancer

MISCELLANEOUS

● Patient access to orphan drugs in France

● The Orphan Drug Act revisited

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