Tratamiento AO

Armstrong AJ, Henke BR, Collado MS, Taylor JM, Pourtaheri TD, Dillberger JE, Roper TD, Wamhoff BR, Olson MW, Figler RA, Hoang SA, Reardon JE, Johns BA.

Identification of 2,2-Dimethylbutanoic Acid (HST5040), a Clinical Development Candidate for the Treatment of Propionic Acidemia and Methylmalonic Acidemia.

J Med Chem. 2021 ;64(8):5037-5048


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Nashabat M, Obaid A, Al Mutairi F, Saleh M, Elamin M, Ahmed H, Ababneh F, Eyaid W, Alswaid A, Alohali L, Faqeih E, Aljeraisy M, Hussein MA, Alasmari A, Alfadhel M.

Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial.

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Yap S, Vara R, Morais A.

Post-transplantation Outcomes in Patients with PA or MMA: A Review of the Literature.

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