Translational medicine. Hôpital Necker Enfants Malades/Université Paris Cité. US FDA and EMA approvals of alpelisib for PROS patients thanks to our works
Paris, France
- Tweets 692
- Following 683
- Followers 1,801
- Likes 1,802
A huge thank you to the @eshgsociety for this extraordinary honor. I am deeply grateful and humbled to receive the Mendel Medal.
This recognition is not mine alone, it belongs to my entire team, whose outstanding work, dedication, and passion made it possible.
Thank you all.
1
17
1,232
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-…
1
8
332
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
Made with AI
1
3
5
276
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-…
3
2
18
932
Our latest paper just released. Congratulations to Ilaria, Charles and the entire team 🍾🥳🔥Oncogenic PIK3CA mutations shape an immunoregulatory microenvironment in mosaic overgrowth disorders academic.oup.com/pnasnexus/a…
1
3
13
743
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…
1
1
6
1,274
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
2
5
615
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
1
8
467
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 🎉🥳🍾🥂
6
34
6,856
Our latest article just released. Congratulations to @IlariaGalasso and @morin_gbrl 🍾🍾🎉🎊Vascular malformations: from genetics to therapeutics | EMBO Molecular Medicine embopress.org/doi/full/10.10…
6
1,245
Our latest review on kidney drug repositioning in @Kidney_Int. Congrats to the 2 outstanding contributors @morin_gbrl and @IsnardPierre1👏🍾🥂Drug repurposing in PIK3CA-related overgrowth spectrum: breakthroughs from overgrowth syndromes to kidney disease kidney-international.org/art…
2
3
472
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
1
6
651
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
2
2
25
1,049
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…
3
4
31
2,159
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.
2
10
1,242
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 👇
2
5
21
1,392
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…
1
5
18
969