Alnylam’s Onpattro is first ever FDA-approved RNAi drug
Onpattro will cost $450,000 a year
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Alnylam’s Onpattro is first ever FDA-approved RNAi drug
https://pharmaphorum.com/news/alnylams-onpattro-first-ever-approved-rnai-drug/
Rare diseases
Spark shares tank after haemophilia gene therapy safety signal
Biotech still pressing on with late stage trials
How challenges like Brexit & insufficient patients hinder Rare Diseases research
Challenges facing Orphan Drugs & Rare Diseases Research
Orphan Drugs & Rare Diseases 2018
Orphan Drugs & Rare Diseases 2018 Conference
NHS does not value CF patients, says Vertex CEO in letter to PM
But Vertex dismisses rumours it will quit the UK
Patient Organisations role in Orphan Drugs clinical development
Orphan Drugs and Rare Diseases Conference – London
