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Results For "rare-disease"

198 News Found

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)


Henlius' Serplulimab granted Orphan-Drug Designation for Small Cell Lung Cancer
Biotech | April 08, 2022

Henlius' Serplulimab granted Orphan-Drug Designation for Small Cell Lung Cancer

The company plans to file its NDA in China and MAA in the EU in 2022, making Hansizhuang potentially to be the world's first PD-1 inhibitor for the first-line treatment of SCLC


ASC Therapeutics gets green light to advance gene therapy for Hemophilia A
Biotech | March 30, 2022

ASC Therapeutics gets green light to advance gene therapy for Hemophilia A

A novel second-generation approach to achieve a safe, durable, and stable transgene expression to overcome the challenges of factor VIII replacement therapies


New spesolimab data show promising results in patients with generalised pustular psoriasis flares
Biotech | March 28, 2022

New spesolimab data show promising results in patients with generalised pustular psoriasis flares

Clinical improvements were consistent across patient subgroups including age, gender, ethnicity, and IL-36 gene mutation status


Clarivate deals report highlights emerging trends in biopharma deal-making
Biotech | March 14, 2022

Clarivate deals report highlights emerging trends in biopharma deal-making

RNA, cell & gene therapies, artificial intelligence, CRISPR and oncology-related deals dominate the landscape