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Results For "rare-disease"

212 News Found

Medison Pharma announces multi-regional agreement with argenx to commercialize Efgartigimod
News | June 08, 2022

Medison Pharma announces multi-regional agreement with argenx to commercialize Efgartigimod

Efgartigimod is approved in the United States as VYVGART for the treatment of adults with generalized myasthenia gravis


Merck KGaA to expand its membrane and filtration manufacturing facility in Ireland
News | May 23, 2022

Merck KGaA to expand its membrane and filtration manufacturing facility in Ireland

The company will invest approximately €440 million to increase membrane manufacturing capacity in Carrigtwohill and to build a new manufacturing facility at Blarney Business Park, both in Cork, Ireland


Soligenix receives US patent allowance for its thermostabilized vaccine platform
Biotech | May 23, 2022

Soligenix receives US patent allowance for its thermostabilized vaccine platform

Includes thermostabilized Ebola vaccines


Don’t be left short-changed post approval – plan in change orders early for accelerated pathways: Stephanie Gaulding
News | May 19, 2022

Don’t be left short-changed post approval – plan in change orders early for accelerated pathways: Stephanie Gaulding

A post approval change order is often necessary, but innovators should plan and prepare for this pre-approval. Planning alongside outsourcing partners will help ensure that only a small-scale study is needed—not a new full clinical study


Takeda launches Adynovate for Hemophilia patients in India
News | May 16, 2022

Takeda launches Adynovate for Hemophilia patients in India

Adynovate in combination with Mypkfit offers personalized treatment and enables HCPs and patients to monitor factor levels from home and help achieve optimum QOL outcomes


Neurocrine Biosciences receives Orphan Drug Designation for Valbenazine
Biotech | May 13, 2022

Neurocrine Biosciences receives Orphan Drug Designation for Valbenazine

The treatment of chorea associated with HD is within the scope of this Orphan Drug Designation


Novo Nordisk and Flagship Pioneering partner to create a portfolio of transformational medicines
Biotech | May 10, 2022

Novo Nordisk and Flagship Pioneering partner to create a portfolio of transformational medicines

Pioneering Medicines and Novo Nordisk Bio Innovation Hub will leverage Flagship Pioneering’s bioplatform companies to create a portfolio of research programmes within cardiometabolic and rare diseases


Genome Insight draws US $ 23 million in Series B Funding
Startup | May 01, 2022

Genome Insight draws US $ 23 million in Series B Funding

Funds will be allocated to accelerate rapid production and high-end curation of whole genome sequences for its clinical application


Danish National Genome Center partners with Lifebit to deliver nationwide personalised medicine
Biotech | April 28, 2022

Danish National Genome Center partners with Lifebit to deliver nationwide personalised medicine

Danish National Genome Center partners with Lifebit to deliver nationwide personalised medicine


Bridge Biotherapeutics enters into an exclusive license agreement with Shaperon
Biotech | April 19, 2022

Bridge Biotherapeutics enters into an exclusive license agreement with Shaperon

By strengthening the company's fibrotic disease development pipeline, Bridge will enhance its strategic focus on the development of comprehensive treatment solutions for idiopathic pulmonary fibrosis (IPF)