Rare diseases Archives - Pharmaphorum

Roche’s SMA drug gets PRIME regulatory status in Europe

https://pharmaphorum.com/news/roches-sma-drug-gets-prime-regulatory-status-in-europe/

2018 in review: How patient centricity led to new drugs that tackle unmet need

https://pharmaphorum.com/views-analysis-patients/2018-in-review-how-patient-centricity-led-to-new-drugs-that-tackle-unmet-need/

Precision Medicine Congress in Berlin on the 10th and 11th of April

https://pharmaphorum.com/events/precision-medicine-congress-in-berlin-on-the-10th-and-11th-of-april/

Novartis/Spark’s sight gene therapy approved in EU

https://pharmaphorum.com/news/novartis-sparks-sight-gene-therapy-approved-in-eu/

FDA approves Sobi’s Gamifant for rare, fatal childhood disease

https://pharmaphorum.com/news/fda-approves-sobis-gamifant-for-rare-fatal-childhood-disease/

Gene therapy firm BioMarin stages haemophilia musical

https://pharmaphorum.com/news/gene-therapy-firm-biomarin-stages-haemophilia-musical/

Novartis says $4m price is reasonable for SMA gene therapy

https://pharmaphorum.com/news/novartis-says-4m-price-is-reasonable-for-sma-gene-therapy/

Alexion joins with Dicerna to develop RNAi therapies for complement diseases

https://pharmaphorum.com/news/alexion-joins-with-dicerna-to-develop-rnai-therapies-for-complement-diseases/

UK biotech Orchard plans $172.5m US IPO to fund rare disease R&D

https://pharmaphorum.com/news/uk-biotech-orchard-plans-172-5m-us-ipo-to-fund-rare-disease-rd/

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