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Results For "rare-diseases"

105 News Found

Emmes launches Cell and Gene Therapy Center
News | December 09, 2022

Emmes launches Cell and Gene Therapy Center

The new center will focus on supporting the clinical trials clients who are developing cell and gene therapies


AstraZeneca receives import and market permission from CDSCO for Dapagliflozin
Drug Approval | November 26, 2022

AstraZeneca receives import and market permission from CDSCO for Dapagliflozin

The receipt of this permission paves way for the launch of Dapagliflozin (Forxiga) tablets of 10 mg in India for the specified additional/expanded indication, subject to the receipt of related statutory approvals


Inceptua Early Access and Sentynl Therapeutics launch early access program for Nulibry for pediatric patients
News | October 19, 2022

Inceptua Early Access and Sentynl Therapeutics launch early access program for Nulibry for pediatric patients

MoCD Type A is a rapidly progressive autosomal recessive inborn error of metabolism resulting in toxic sulfite levels causing neurologic sequelae


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
Diagnostic Center | 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