Novartis publishes long-term data from SMA gene therapyNovartis has produced data demonstrating its Zolgensma gene therapy produces long term benefits in the rare muscle wasting Share XNovartis publishes long-term data from SMA gene therapyhttps://pharmaphorum.com/news/novartis-publishes-long-term-data-from-sma-gene-therapy/
Genetic testing hopes to speed up drug development in rare eye diseasesWhen it comes to fighting rare diseases, the biggest barrier to overcome is often the lack of knowledge Share XGenetic testing hopes to speed up drug development in rare eye diseaseshttps://pharmaphorum.com/r-d/views-analysis-r-d/genetic-testing-hopes-to-speed-up-drug-development-in-rare-eye-diseases/
Understanding the impact of a rare disease – the Health Heroes podcastEpisode one of Kantar’s Health Heroes podcast looks at caregiving challenges for Gaucher disease and how patient registries Share XUnderstanding the impact of a rare disease – the Health Heroes podcasthttps://pharmaphorum.com/podcast/health-heroes-podcast-rare-disease-impact/
AI for rare diseases: putting tech to work for small populationsWith the 12th annual Rare Disease Day on the horizon, it’s a pertinent time to think about how Share XAI for rare diseases: putting tech to work for small populationshttps://pharmaphorum.com/spotlight/ai-in-pharma-marketing/ai-rare-diseases/
The biggest challenges facing rare disease pharma – and how the industry can tackle themAs Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers Share XThe biggest challenges facing rare disease pharma – and how the industry can tackle themhttps://pharmaphorum.com/r-d/views-analysis-r-d/biggest-challenges-rare-disease/
Podcast series to spotlight health heroes launched by KantarA new pharmaceutical podcast putting health heroes in the spotlight debuts in the runup to Rare Diseases Day Share XPodcast series to spotlight health heroes launched by Kantarhttps://pharmaphorum.com/news/podcast-series-to-spotlight-health-heroes-launched-by-kantar/
Discover who’s speaking at Real-World Evidence 2020: Rare Diseases and Innovative TherapiesDon’t miss your chance to attend: Real-World Evidence 2020: Rare Diseases and Innovative Therapies 1–2 April 2020 1 America Share XDiscover who’s speaking at Real-World Evidence 2020: Rare Diseases and Innovative Therapieshttps://pharmaphorum.com/events/discover-whos-speaking-at-real-world-evidence-2020-rare-diseases-and-innovative-therapies/
Millendo’s Julia Owens on leadership in rare diseasesRare disease specialists Millendo Therapeutics are hoping their drug livoletide will become the first treatment for Prader-Willi syndrome. Share XMillendo’s Julia Owens on leadership in rare diseaseshttps://pharmaphorum.com/r-d/views-analysis-r-d/millendo-julia-owens-leadership-rare-diseases/
Chiesi starts rare diseases division, ahead of potential Fabry disease filingItalian pharma Chiesi has begun a new rare diseases division as it builds towards a potential commercial launch Share XChiesi starts rare diseases division, ahead of potential Fabry disease filinghttps://pharmaphorum.com/news/chiesi-starts-rare-diseases-division-as-fabry-treatment/
