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Tag: Rare disease

rare diseases

17/02/2020 7 mins

How Can We Improve the Diagnosis and Treatment of Rare Diseases?

lysosomal storage disorders azafaros rare disease

10/02/2020 2 mins

First-in-Class Drug For Inherited Metabolic Diseases Funded with €25M

brummelkamp Scenic Biotech

05/02/2020 5 mins

Harnessing Genetic Suppression to Treat Rare Disease

chemocentryx vifor fresenius medical care renal pharma anca vasculitis

26/11/2019 2 mins

Rare Inflammatory Disease Drug Beats Immunosuppressants in Phase III

mereo biopharma brittle bone disease osteogenesis imperfecta

11/11/2019 2 mins

Mereo BioPharma’s Brittle Bone Disease Drug Stumbles in Phase II

centogene rare diseases diagnostic ipo nasdaq

07/11/2019 1 min

€51M Nasdaq IPO Will Fund Centogene’s Rare Disease Diagnostics

RNAi coming of age

28/10/2019 6 mins

RNAi: A Coming of Age Story

ai healx rare disease

16/10/2019 2 mins

UK Firm Raises €51M to Repurpose Drugs for Rare Diseases Using AI

khondrion netherlands melas syndrome inherited diseases

20/09/2019 2 mins

This Biotech Targets Mitochondria to Treat Inherited Diseases

pharvaris hereditary angioedema firazyr

11/09/2019 1 min

Dutch Company Raises €60M to Treat Inherited Inflammatory Disease

novartis avexis zolgensma fda data manipulation

07/08/2019 2 mins

FDA Considers Imposing Penalties on Novartis over Data Manipulation

bluebird bio gene therapy beta thalassemia

04/06/2019 2 mins

First Gene Therapy for Beta Thalassemia Approved in Europe

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