Rare diseases

FDA backs second rare disease drug from Ultragenyx in the space of a fortnight

https://pharmaphorum.com/news/fda-backs-ultragenyxs-second-rare-disease-drug-in-the-space-of-a-fortnight/

Registration opens for SMi’s 4th Annual Ophthalmic Drugs Conference

https://pharmaphorum.com/partner-content/registration-opens-for-smis-4th-annual-ophthalmic-drugs-conference/

Sarepta agree gene therapy R&D pact with Selecta

https://pharmaphorum.com/news/sarepta-forges-gene-therapy-rd-alliance-with-selecta/

Alnylam’s shares dip as analyst backs rival after rare disease data reveal

https://pharmaphorum.com/news/alnylams-shares-dip-as-analyst-backs-rival-after-rare-disease-data-reveal/

Calliditas hits $90m jackpot as biotech IPO craze continues

https://pharmaphorum.com/news/calliditas-hits-90m-jackpot-as-biotech-ipo-craze-continues/

Envisagenics presents latest case study at Therapeutics and Delivery

https://pharmaphorum.com/partner-content/envisagenics-presents-latest-case-study-at-therapeutics-and-delivery/

Patient insights: Neuromyelitis optica

https://pharmaphorum.com/views-analysis-patients/patient-insights-neuromyelitis-optica/

Novartis offers reimbursement options as EU backs costly SMA therapy

https://pharmaphorum.com/news/novartis-reimbursement-options-as-eu-approves-ultra-pricey-sma-drug/

Takeda posts surprise profit a year after Shire mega-merger

https://pharmaphorum.com/news/takeda-posts-surprise-profit-a-year-after-shire-mega-merger/

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