Translational medicine. Hôpital Necker Enfants Malades/Université Paris Cité. US FDA and EMA approvals of alpelisib for PROS patients thanks to our works

Joined June 2018
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Guillaume Canaud (MD, PhD) retweeted
Big news today: Vijoice (Alpelisib) has been approved for use by the EMA. This medication addresses an unmet medical need for patients with severe or life-threatening PIK3CA-related overgrowth spectrum (PROS) disorders ema.europa.eu/en/news/first-…
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Guillaume Canaud (MD, PhD) retweeted
First Drug for Severe Overgrowth Disorders Approved in Europe! The European Medicines Agency has conditionally authorized the use of Vijoice (aka alpelisib/Novartis) for the treatment of patients with PROS disorders. PROS = PIK3CA-related overgrowth spectrum. A diverse group of rare genetic conditions that are characterized by uncontrolled growth of some tissues. They are caused by a mutation in the PIK3CA gene. Vijoice is indicated for patients over two years of age with severe or life-threatening manifestations of PROS, requiring systemic therapy. Vijoice showed a 20% tumor reduction in about 37.5% of patients. This is a condition that has many unmet medical needs, and this is the first step in the right direction. #PROS #GrowthDisorder #EMA #EuropeanMedicine
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Following US FDA approval in April 2022, our group has reached another major milestone today with the EMA conditional approval of alpelisib for patients with severe and life-threatening PIK3CA-related overgrowth spectrum (PROS). Great news for EU patients ema.europa.eu/en/news/first-…
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What extraordinary news — I’m extremely proud and honored. Thank you @eshgsociety for this very prestigious award, which I share with the entire team. 🎉🍾🥂
The ESHG Award, ELPAG Award and the Mendel Lecturers of the #eshg2026 #hybridconference have talked to Mary Rice. Read the interviews and find out more about their backgrounds: 2026.eshg.org/speakers/speak…
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Bravo @casanova_lab So well deserved 🍾
We're proud to announce that infectious disease pioneer Jean-Laurent Casanova, MD, PhD, of @RockefellerUniv, @InstitutImagine, @hhmi_science, & @Casanova_Lab, is the distinguished recipient of the 2026 Mechthild Esser #NemmersPrize in Medical Science. 🔗spr.ly/6017hCLtR
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Merci
📢Journée mondiale des maladies rares | Focus sur le syndrome de Cloves avec @CanaudLab, docteur au sein de l’unité de Médecine translationnelle et thérapies ciblées @hopital_necker 👉 swll.to/u3rtlvF Cc @gouvernementFR @Sante_Gouv @SantePubliqueFr
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Aujourd’hui nous célébrions les 10 ans d’introduction de l’alpelisib (BYL719) chez Emmanuel, le premier patient PIK3CA traité dans le monde. Immense fierté🇫🇷 10 years on alpelisib for Emmanuel, the first ever PROS patient treated. Fantastic accomplishment for all of us 🎉🥳🍾🥂
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Guillaume Canaud (MD, PhD) retweeted
Wonderful team🙏🍾❤️
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Guillaume Canaud (MD, PhD) retweeted
#France2030 | Félicitations à Guillaume Canaud @CanaudLab, lauréat d’une Chaire d’excellence en Biologie/Santé pour son projet sur les malformations vasculaires liées à KRAS. 🎯Objectif : développer des thérapies ciblées pour une médecine ultra personnalisée. 👏🏼
📢#France2030 15 nouveaux chercheurs et chercheuses d'exception récompensés pour les « Chaires d’excellence en Biologie / Santé » !🧑‍🔬 Ce dispositif vise à renforcer l’attractivité de la recherche biomédicale française🧪🇫🇷🥼 Pour découvrir les lauréats👉swll.to/ilGDHI
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Excellente nouvelle et merci pour votre soutien! Ce financement devrait accélérer l’identification de nouveaux traitements pour les malformations vasculaires secondaires a des mutations de KRAS 🍾🎊🥳 Wonderful news! This is a great funding for KRAS-vascular malformations! 🙏🙏
📢#France2030 15 nouveaux chercheurs et chercheuses d'exception récompensés pour les « Chaires d’excellence en Biologie / Santé » !🧑‍🔬 Ce dispositif vise à renforcer l’attractivité de la recherche biomédicale française🧪🇫🇷🥼 Pour découvrir les lauréats👉swll.to/ilGDHI
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Guillaume Canaud (MD, PhD) retweeted
Here is our latest publication. Somatic PIK3R1 mutations in patients and treatment with alpelisib. Congratulations to the whole team and particularly to two very talented students @morin_gbrl and @Alexandre Garneau🥳🥂🍾 EMBO Molecular Medicine embopress.org/doi/full/10.10…
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Guillaume Canaud (MD, PhD) retweeted
Key messages: 1-No fertility issues in 3 PROS patients with prolonged exposure to alpelisib 2-No embryotoxicity 3-Unfortunately, we observed disease recurrence following alpelisib withdrawal 4-But, no resistance to drug when alpelisib was reintroduced after pregnancies.
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Guillaume Canaud (MD, PhD) retweeted
Happy to share our latest article: « First report of successful pregnancies after treatment with alpelisib for PIK3CA-related overgrowth spectrum » Congratulations to the whole team and particularly to @morin_gbrl 🥳🍾🎉 @ejhg_journal rdcu.be/epJZ1 Key messages 👇

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Guillaume Canaud (MD, PhD) retweeted
Our latest paper is out today in @PNAS. Congratulations to Quitterie, Sophia, Marina, and the entire team!🍾🤩🥳PIK3CA gain-of-function mutation in Schwann cells leads to severe neuropathy and aerobic glycolysis through a non-cell autonomous effect | PNAS pnas.org/doi/10.1073/pnas.24…
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Guillaume Canaud (MD, PhD) retweeted
[PUBLICATION] First report of successful pregnancies after treatment with alpelisib for PIK3CA-related overgrowth spectrum. ➡️Fertility preserved ➡️No embryotoxicity ➡️Disease remained responsive after treatment pause 📖Details here: nature.com/articles/s41431-0…
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