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Results For "rare-disease"

198 News Found

Emmes acquires Casimir, its fourth major acquisition
Biotech | March 11, 2022

Emmes acquires Casimir, its fourth major acquisition

Casimir further differentiates Emmes’ industry-leading rare disease research capabilities


Zydus Lifesciences subsidiary Sentynl acquires BridgeBio’s Nulibry
Biotech | March 09, 2022

Zydus Lifesciences subsidiary Sentynl acquires BridgeBio’s Nulibry

Nulibry is approved by the U.S. Food and Drug Administration (USFDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening paediatric genetic disorder


Suvoda unveils new brand to reflect expanded focus in complex clinical trial management
Biotech | March 08, 2022

Suvoda unveils new brand to reflect expanded focus in complex clinical trial management

Alongside brand refresh, the leading clinical trial technology company announces eConsent and eCOA offerings to better support the patient clinical trial journey


AstraZeneca and Neurimmune enter licencing agreement to develop and commercialise NI006
Biotech | March 01, 2022

AstraZeneca and Neurimmune enter licencing agreement to develop and commercialise NI006

Under the terms of the agreement, the upfront payment from Alexion to Neurimmune is US $ 30m


GliaPharm SA collaborate with Wyss Centre for Alzheimer therapy
Biotech | February 25, 2022

GliaPharm SA collaborate with Wyss Centre for Alzheimer therapy

The collaboration will accelerate the testing of GliaPharm’s lead molecules in preclinical models of Alzheimer’s disease and further the development of neuroimaging biomarkers


Biohaven licenses Taldefgrobep Alfa from Bristol Myers Squibb
Biotech | February 25, 2022

Biohaven licenses Taldefgrobep Alfa from Bristol Myers Squibb

It is a Phase 3 ready anti-myostatin adnectin for Spinal Muscular Atrophy (SMA).


ValGenesis selected for validating French pharma firm
Digitisation | February 18, 2022

ValGenesis selected for validating French pharma firm

With ValGenesis VLMS, change control driven validation activities are reduced from weeks to mere hours, and the company gains faster access to detailed metrics that help reduce costs and cycle time


Moderna announces expansion in key Asian markets
News | February 16, 2022

Moderna announces expansion in key Asian markets

New subsidiaries to be established in Malaysia, Taiwan, Singapore, and Hong Kong


Sanofi unveils new corporate brand and logo
News | February 05, 2022

Sanofi unveils new corporate brand and logo

Sanofi unveiled a new bold and unifying corporate brand that supports the modernization and transformation the company launched in December 2019


Eplontersen granted Orphan Drug Designation in the US
Biotech | January 24, 2022

Eplontersen granted Orphan Drug Designation in the US

The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US