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Results For "rare-diseases"

142 News Found

CHMP recommends conditional marketing authorization for Spesolimab for generalized pustular psoriasis flares
News | October 17, 2022

CHMP recommends conditional marketing authorization for Spesolimab for generalized pustular psoriasis flares

CHMP positive opinion is based on evidence from the EFFISAYIL trial, the largest clinical trial in


BioMarin simplifies organizational structure to increase efficiency
News | October 09, 2022

BioMarin simplifies organizational structure to increase efficiency

The reduction in force will result in financial savings of approximately $50 million annually beginning in 2023


South Korea nextgen sequencing tests market to grow at 4% CAGR during 2022-2030: GlobalData
Clinical Trials | September 28, 2022

South Korea nextgen sequencing tests market to grow at 4% CAGR during 2022-2030: GlobalData

There is also a growing interest in whole generation sequencing (WGS)


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