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Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial.
Di Rocco M, Forleo-Neto E, Pignolo RJ, Keen R, Orcel P, Funck-Brentano T, Roux C, Kolta S, Madeo A, Bubbear JS, Tabarkiewicz J, Szczepanek M, Bachiller-Corral J, Cheung AM, Dahir KM, Botman E, Raijmakers PG, Al Mukaddam M, Tile L, Portal-Celhay C, Sarkar N, Hou P, Musser BJ, Boyapati A, Mohammadi K, Mellis SJ, Rankin AJ, Economides AN, Trotter DG, Herman GA, O'Meara SJ, DelGizzi R, Weinreich DM, Yancopoulos GD, Eekhoff EMW, Kaplan FS. Di Rocco M, et al. Nat Med. 2023 Oct;29(10):2615-2624. doi: 10.1038/s41591-023-02561-8. Epub 2023 Sep 28. Nat Med. 2023. PMID: 37770652 Free PMC article. Clinical Trial.
GNAO1 Haploinsufficiency: The Milder End of the GNAO1 Phenotypic Spectrum.
Galosi S, Novelli M, Di Rocco M, Flex E, Messina E, Pollini L, Parrini E, Pisani F, Guerrini R, Leuzzi V, Martinelli S. Galosi S, et al. Among authors: di rocco m. Mov Disord. 2023 Dec;38(12):2313-2314. doi: 10.1002/mds.29585. Epub 2023 Aug 26. Mov Disord. 2023. PMID: 37632268 No abstract available.
GAU-PED study for early diagnosis of Gaucher disease in children with splenomegaly and cytopenia.
Pession A, Di Rocco M, Venturelli F, Tappino B, Morello W, Santoro N, Giordano P, Filippini B, Rinieri S, Russo G, Girardi K, Ruggiero A, Galea E, Antonucci R, Tovaglieri N, Porta F, Tartaglione I, Giona F, Fagioli F, Burlina A; Pediatric Gaucher Study Group. Pession A, et al. Among authors: di rocco m. Orphanet J Rare Dis. 2023 Jun 16;18(1):151. doi: 10.1186/s13023-023-02760-z. Orphanet J Rare Dis. 2023. PMID: 37328863 Free PMC article.
The recurrent pathogenic Pro890Leu substitution in CLTC causes a generalized defect in synaptic transmission in Caenorhabditis elegans.
Pannone L, Muto V, Nardecchia F, Di Rocco M, Marchei E, Tosato F, Petrini S, Onorato G, Lanza E, Bertuccini L, Manti F, Folli V, Galosi S, Di Schiavi E, Leuzzi V, Tartaglia M, Martinelli S. Pannone L, et al. Among authors: di rocco m. Front Mol Neurosci. 2023 May 31;16:1170061. doi: 10.3389/fnmol.2023.1170061. eCollection 2023. Front Mol Neurosci. 2023. PMID: 37324589 Free PMC article.
300 results