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Current insights in ultra-rare adenylosuccinate synthetase 1 myopathy - meeting report on the First Clinical and Scientific Conference. 3 June 2024, National Centre for Advancing Translational Science, Rockville, Maryland, the United States of America.
Rybalka E, Park HJ, Nalini A, Baskar D, Polavarapu K, Durmus H, Xia Y, Wan L, Shieh PB, Moghadaszadeh B, Beggs AH, Mack DL, Smith AST, Hanna-Rose W, Jinnah HA, Timpani CA, Shen M, Upadhyay J, Brault JJ, Hall MD, Baweja N, Kakkar P. Rybalka E, et al. Among authors: timpani ca. Orphanet J Rare Dis. 2024 Nov 26;19(1):438. doi: 10.1186/s13023-024-03429-x. Orphanet J Rare Dis. 2024. PMID: 39593137 Free PMC article.
Dimethyl fumarate modulates the dystrophic disease program following short-term treatment.
Timpani CA, Kourakis S, Debruin DA, Campelj DG, Pompeani N, Dargahi N, Bautista AP, Bagaric RM, Ritenis EJ, Sahakian L, Debrincat D, Stupka N, Hafner P, Arthur PG, Terrill JR, Apostolopoulos V, de Haan JB, Guven N, Fischer D, Rybalka E. Timpani CA, et al. JCI Insight. 2023 Nov 8;8(21):e165974. doi: 10.1172/jci.insight.165974. JCI Insight. 2023. PMID: 37751291 Free PMC article.
Adenylosuccinic Acid: An Orphan Drug with Untapped Potential.
Rybalka E, Kourakis S, Bonsett CA, Moghadaszadeh B, Beggs AH, Timpani CA. Rybalka E, et al. Among authors: timpani ca. Pharmaceuticals (Basel). 2023 May 31;16(6):822. doi: 10.3390/ph16060822. Pharmaceuticals (Basel). 2023. PMID: 37375769 Free PMC article.
30 results