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Correction to: short and long-term acceptability and efficacy of extended-release cornstarch in the hepatic glycogen storage diseases: results from the Glyde study.
Weinstein DA, Jackson RJ, Brennan EA, Williams M, Davison JE, de Boer F, Derks T, Ellerton C, Faragher B, Gribben J, Labrune P, McKittrick KM, Murphy E, Ross KM, Steuerwald U, Voillot C, Woodward A, Mundy HR. Weinstein DA, et al. Orphanet J Rare Dis. 2024 Aug 30;19(1):317. doi: 10.1186/s13023-024-03305-8. Orphanet J Rare Dis. 2024. PMID: 39215332 Free PMC article. No abstract available.
Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis.
Quadrini KJ, Vrentas C, Duke C, Wilson C, Hinderer CJ, Weinstein DA, Al-Zaidy SA, Browne SE, Wilson JM, Ni YG. Quadrini KJ, et al. Among authors: weinstein da. Mol Ther Methods Clin Dev. 2024 Aug 10;32(3):101318. doi: 10.1016/j.omtm.2024.101318. eCollection 2024 Sep 12. Mol Ther Methods Clin Dev. 2024. PMID: 39282076 Free PMC article.
Short and long-term acceptability and efficacy of extended-release cornstarch in the hepatic glycogen storage diseases: results from the Glyde study.
Weinstein DA, Jackson RJ, Brennan EA, Williams M, Davison JE, Boer F, Derks T, Ellerton C, Faragher B, Gribben J, Labrune P, McKittrick KM, Murphy E, Ross KM, Steuerwald U, Voillot C, Woodward A, Mundy HR. Weinstein DA, et al. Orphanet J Rare Dis. 2024 Jul 9;19(1):258. doi: 10.1186/s13023-024-03274-y. Orphanet J Rare Dis. 2024. PMID: 38982397 Free PMC article. Clinical Trial.
Glycogen Storage Disease type IA refractory to cornstarch: Can next generation sequencing offer a solution?
Steg Saban O, Pode-Shakked B, Abu-Libdeh B, Granot M, Barkai G, Haberman Y, Roterman I, Lahad A, Shouval DS, Weiss B, Marek-Yagel D, Barel O, Loberman-Nachum N, Abraham S, Somech R, Weinstein DA, Anikster Y. Steg Saban O, et al. Among authors: weinstein da. Eur J Med Genet. 2022 Jun;65(6):104518. doi: 10.1016/j.ejmg.2022.104518. Epub 2022 May 9. Eur J Med Genet. 2022. PMID: 35550444
112 results