Etuvetidigene Autotemcel for the Treatment of Wiskott–Aldrich Syndrome
Francesca Ferrua, Sabina Cenciarelli, Stefania Giannelli, Stefania Galimberti, Shanmuganathan Chandrakasan, Federico Fraschetta, Carmen Caputo, Davide Sala, Ilaria Monti, Federica Barzaghi, Valeria Calbi, Daniele Canarutto, Giulia Consiglieri, Matteo Doglio, Francesca Fumagalli, Vera Gallo, Maddalena Migliavacca, Salvatore Marco Recupero, Francesca Tucci, Alessia Orsini, Raffaella Milani, Mariam Datukishvili, Simona De Gregori, Eugenio Montini, Paolo Silvani, Matías Soncini, Elena Tomasetto, Koen van Rossem, Laura Castagnaro, Federica Miotto, Stefano Zancan, Celeste Scotti, Sean Russell, Luigi Naldini, Fabio Ciceri, Maria Ester Bernardo, Suhag H Parikh, Maria Pia Cicalese, Alessandro Aiuti
In the authors' words
This journal does not let us republish the full abstract. Here are the two sentences Pipette selected, quoted from it. Read the rest at the publisher.
CONCLUSIONS: Our results show that the effects of etu-cel are consistent with a sustained clinical benefit in persons with Wiskott-Aldrich syndrome.
Appeared: Friday, September 25. New England Journal of Medicine. Peer-reviewed journal.
More on Treatments & drugs that day
Drug repurposing identifies candidate uterus-selective tocolytics for preterm labor
This approach established a systematic strategy for identifying uterus-selective tocolytics, and these findings support further preclinical development of repurposed drugs and drug combinations for the treatment of preterm labor to improve pregnancy and neonatal outcomes.
Shajila Siricilla, Christopher J. Hansen, Jeremy Staub and 18 more — Science Translational Medicine
Peer-reviewed journalReal-world useThe nucleoside analog kamuvudine-9 shows protective and therapeutic efficacy in a mouse model of multiple sclerosis
Here, we report that in the experimental autoimmune encephalitis mouse model of MS, treatment with kamuvudine-9 (K-9), an NRTI derivative with an enhanced safety profile, prevented further neurological deficits and reversed preexisting paralysis and vision loss.
Praveen Yerramothu, Kameshwari Ambati, Joseph Magagnoli and 36 more — Science Translational Medicine
Peer-reviewed journalReal-world useA design approach for bitopic kinase inhibitors
PonatiLink-2 outperforms clinically relevant treatments in mouse models of BCR :: ABL1 -driven cancer, both in combination with dasatinib against wild-type BCR :: ABL1 and as a single agent against ponatinib-resistant disease.
Jack W. Stevenson, Kevin Lou, Joshua A. Reynolds and 25 more — Nature
Peer-reviewed journalReal-world useA Single Suprachoroidal Injection of AXT107 (Gersizangitide), a Multimodal, Long-Acting Integrin-Disrupting Peptide, in Patients with Neovascular Age-Related Macular Degeneration: Results of the Phase 1/2a DISCOVER Trial
Conclusions In this first-in-human study, a single suprachoroidal injection of AXT107 up to 0.5 mg was well tolerated through Week 40, with no drug-related serious adverse events, no clinically significant intraocular inflammation, and no sustained IOP elevations.
N. B. Pandey, A. C. Mirando, D. R. P. Almeida and 6 more — medRxiv
PreprintReal-world usePhotoinitiator-free gelatin hydrogel microneedles for acute gouty arthritis treatment
Overall, this initiator-free, photo-crosslinkable GelLA-based MNs system offered a safe, efficient, and user-friendly strategy for local drug delivery in the management of acute gouty arthritis.
Jianhua Zhang, Hengjie Zhang, Tianyou Wang and 6 more — Science Advances
Peer-reviewed journalReal-world use