inherited disease
Applied Therapeutics slumps as FDA seeks more data on lead drug
Phil Taylor
Applied Therapeutics, galactosaemia, inherited disease, rare disease, regulatory
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Applied Therapeutics slumps as FDA seeks more data on lead drug
https://pharmaphorum.com/news/applied-therapeutics-slumps-as-fda-seeks-more-data-on-lead-drug/
Articles/ Rare diseases/ Views and analysis
Out of the darkness: new gene therapies for eye diseases
Linda Banks
eye disease, gene therapy, inherited disease, patient perspective, rare disease, Spark Therapeutics
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US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.
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Out of the darkness: new gene therapies for eye diseases
https://pharmaphorum.com/views-and-analysis/gene-therapies-eye-diseases/