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Trend Chart on Orphans – February 13th, 2020
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Trend Chart on Orphans – February 13th, 2020

Trend Chart on Orphans – February 15th, 2020

FEATURE STORY
● The potential of gene therapy for mucopolysaccharidosis type I
GENE THERAPY
● Repair of retinal degeneration following ex vivo Minicircle DNA gene therapy and transplantation of corrected photoreceptor progenitors
RARE DISEASES
● Genetics and other omics in pediatric pulmonary arterial hypertension
● Natural history of adult patients with GM2 gangliosidosis
● Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine
CLINICAL TRIALS – DATA
● Lentiviral gene therapy for X-linked chronic granulomatous disease
INDUSTRIAL LANDSCAPE & AGREEMENTS
● Lonza supporting Rocket Pharma with clinical manufacturing of RP-L201 for the treatment of Leukocyte Adhesion Deficiency-I
● Bayer application for nifurtimox to treat pediatric patients with Chagas disease submitted for approval in US
MISCELLANEOUS
● Towards European harmonisation of healthcare for patients with rare immune disorders
● Use of real-world evidence in regulatory decisions for rare diseases in the United States-Current status and future directions

To read the full articles, click here: Trend Chart on Orphans – February 13

 


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