Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth
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
It was recognized for its holistic initiatives designed to improve the quality of life and standards of care for patients
It is the first integrated digital PCR solution ideal for oncology, cell and gene therapy development
It’s the company’s fourth manufacturing site worldwide. Others are located in the US, UK and China
Tezepelumab has been granted Orphan Drug Designation (ODD) in the US by the U.S. Food and Drug Administration (FDA) for the treatment of eosinophilic esophagitis (EoE)
Extension of hypericin orphan designation beyond cutaneous T-cell lymphoma
The drug is also intended for the treatment of glioblastoma multiforme
It is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and could be eligible for treatment with Voxzogo
Seen in 1 in 3 million births, the malformation was detected at the antenatal stage during the mother's pregnancy
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