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Results For "rare-diseases"

109 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


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


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