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Results For "rare-disease"

198 News Found

Pfizer and OPKO update on the Biologics License Application for Somatrogon
Biotech | January 22, 2022

Pfizer and OPKO update on the Biologics License Application for Somatrogon

Pfizer is evaluating the FDA’s comments and will work with the agency to determine an appropriate path forward


Veristat supported marketing applications for 12 % of all FDA Novel Drug Approvals in 2021
News | January 12, 2022

Veristat supported marketing applications for 12 % of all FDA Novel Drug Approvals in 2021

This surpasses the number of approvals supported in 2020


Pfizer and Beam enter research collaboration for In Vivo base editing programme
Biotech | January 11, 2022

Pfizer and Beam enter research collaboration for In Vivo base editing programme

Four-year research collaboration combines Pfizer’s deep experience in global drug development, including programs utilizing messenger RNA (mRNA), lipid nanoparticles (LNP), and gene therapy, with Beam’s leadership in base editing and mRNA/LNP delivery technologies


Clarivate identifies seven potential blockbuster drugs in Annual Drugs to Watch report
News | January 10, 2022

Clarivate identifies seven potential blockbuster drugs in Annual Drugs to Watch report

Despite the challenges of pandemic disruptions, drug developers advance promising therapies for conditions, including Alzheimer's, diabetes and asthma


AstraZeneca and Neurimmune to develop and commercialise NI006
Biotech | January 07, 2022

AstraZeneca and Neurimmune to develop and commercialise NI006

Investigational human monoclonal antibody in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy, a systemic, progressive and fatal condition


Roche completes acquisition of TIB Molbiol
Medical Device | December 01, 2021

Roche completes acquisition of TIB Molbiol

TIB Molbiol excels in ultra-rapid assay development for emerging infectious diseases, strongly demonstrated during the Covid-19 pandemic


Aadi Bioscience secures U.S FDA approval of its first product Fyarro
Drug Approval | November 26, 2021

Aadi Bioscience secures U.S FDA approval of its first product Fyarro

Fyarro is the first and only approved therapy for adults for the treatment of malignant PEComa in adults


Novo Nordisk to acquire Dicerna Pharmaceuticals for US $ 3.3 bn
Biotech | November 20, 2021

Novo Nordisk to acquire Dicerna Pharmaceuticals for US $ 3.3 bn

Dicerna develops RNAi-based therapies to selectively silence genes that cause or contribute to disease


U.S. FDA approves first drug to improve growth in kids with most common form of dwarfism
Drug Approval | November 20, 2021

U.S. FDA approves first drug to improve growth in kids with most common form of dwarfism

Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth


World’s first Whole Genome Sequencing study uncovers new and potential therapies
News | November 12, 2021

World’s first Whole Genome Sequencing study uncovers new and potential therapies

The ground-breaking project, led by Genomics England and NHS England, was established in 2013 to sequence 100,000 whole genomes from NHS patients and their families