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Results For "rare-diseases"

109 News Found

BridgeBio Pharma, Sentynl Therapeutics receive marketing authorization in the EU for fosdenopterin
Drug Approval | September 22, 2022

BridgeBio Pharma, Sentynl Therapeutics receive marketing authorization in the EU for fosdenopterin

NULIBRY is a first-in-class cPMP substrate replacement therapy to reduce the risk of mortality in patients with MoCD Type A.


Cytel unlocks innovative clinical development expertise for APAC biopharma sector
Biotech | September 15, 2022

Cytel unlocks innovative clinical development expertise for APAC biopharma sector

Cytel has now established entities in Australia, Shanghai, Beijing, and Singapore, and plans to expand into Seoul and Tokyo in the coming years.


Funds for R&D in drug discovery and manufacturing :PIB
Policy | July 26, 2022

Funds for R&D in drug discovery and manufacturing :PIB

An outlay of Rs. 1,500 crore has been approved for strengthening/ up-gradation of these NIPERs for the period 2021-22 to 2025-26.


H Clinical adds commercial home health capabilities for pharma industry
News | July 01, 2022

H Clinical adds commercial home health capabilities for pharma industry

H Clinical is the leader in decentralized clinical trial support and home visits in Latin America


DTx Pharma appoints Peter Condon as Chief Business Officer
People | June 29, 2022

DTx Pharma appoints Peter Condon as Chief Business Officer

Condon joins DTx Pharma with more than 20 years of experience in business development, commercial planning, and operations.


Syntegra partners with TMA Precision to reduce the diagnostic odyssey for rare disease with synthetic data-enabled tools
Digitisation | June 16, 2022

Syntegra partners with TMA Precision to reduce the diagnostic odyssey for rare disease with synthetic data-enabled tools

TMA will leverage Syntegra's synthetic data engine to accelerate the building of predictive diagnostic algorithms


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


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