Rare diseases

SMi’s 9th Annual Orphan Drugs & Rare Diseases Conference

https://pharmaphorum.com/events/smis-9th-annual-orphan-drugs-rare-diseases-conference/

NHS chief hails SMA drug deal – and was that a swipe at Vertex?

https://pharmaphorum.com/news/nhs-chief-hails-sma-drug-deal-and-was-that-a-swipe-at-vertex/

Registration Opens for SMi’s 9th Annual Orphan Drugs & Rare Diseases Conference

https://pharmaphorum.com/partner-content/registration-opens-for-smis-9th-annual-orphan-drugs-rare-diseases-conference/

Catalyst shares dive amid speculation about ‘off-label’ rare disease rival

https://pharmaphorum.com/news/catalyst-shares-dive-amid-speculation-about-off-label-rare-disease-rival/

Orphan medicines launch excellence: sustaining launch success

https://pharmaphorum.com/market-access-2/orphan-medicines-launch-excellence-sustaining-launch-success/

UK biotech Mereo gears up for key brittle bone drug data

https://pharmaphorum.com/news/uk-biotech-mereo-gears-up-for-key-brittle-bone-drug-data/

https://pharmaphorum.com/news/nice-backs-akceas-tegsedi-pipping-alnylam-to-hattr-okay/

Alnylam to file gene-silencing porphyria drug

https://pharmaphorum.com/news/alnylam-to-file-gene-silencing-porphyria-drug/

4th Annual Antibody Drug Conjugates (ADC) Summit: From Target to Therapeutics

https://pharmaphorum.com/partner-content/4th-annual-antibody-drug-conjugates-adc-summit-from-target-to-therapeutics/

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