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Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705.
N Engl J Med. 2022.
PMID: 36129998
Free article.
Clinical Trial.
A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood.
Hainque E, Caillet S, Leroy S, Flamand-Roze C, Adanyeguh I, Charbonnier-Beaupel F, Retail M, Le Toullec B, Atencio M, Rivaud-Péchoux S, Brochard V, Habarou F, Ottolenghi C, Cormier F, Méneret A, Ruiz M, Doulazmi M, Roubergue A, Corvol JC, Vidailhet M, Mochel F, Roze E.
Hainque E, et al. Among authors: retail m.
Orphanet J Rare Dis. 2017 Oct 2;12(1):160. doi: 10.1186/s13023-017-0713-2.
Orphanet J Rare Dis. 2017.
PMID: 28969699
Free PMC article.
Clinical Trial.
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