Rare diseases Archives -

Rare diseases, repurposing and the role of AI

https://pharmaphorum.com/r-d/views-analysis-r-d/rare-diseases-repurposing-and-the-role-of-ai/

Flying the flag for research in Aarskog syndrome

https://pharmaphorum.com/views-analysis-patients/flying-the-flag-for-research-in-aarskog-syndrome/

CAR-T Beyond Oncology Digital Summit

https://pharmaphorum.com/events/car-t-beyond-oncology-digital-summit/

Gilead pays $97m to resolve US illegal kickback claims

https://pharmaphorum.com/news/gilead-pays-97m-to-resolve-us-illegal-kickback-claims/

Chasing Janssen, Alexion and Caelum start phase 3 AL amyloidosis trial

https://pharmaphorum.com/news/chasing-janssen-alexion-and-caelum-start-phase-3-al-amyloidosis-trial/

Janssen files Darzalex Faspro for rare disease light chain amyloidosis

https://pharmaphorum.com/news/janssen-files-new-darzalex-for-rare-disease-light-chain-amyloidosis/

Searching for answers in rare epilepsy

https://pharmaphorum.com/views-analysis-patients/searching-for-answers-in-rare-epilepsy/

BioMarin files achondroplasia drug after rejection for haemophilia therapy

https://pharmaphorum.com/news/biomarin-files-achondroplasia-drug-after-rejection-for-haemophilia-therapy/

Healx uses AI to find drugs for Angelman Syndrome

https://pharmaphorum.com/news/healx-uses-ai-to-find-drugs-for-angelman-syndrome/

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