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Publications and Presentations

Publications and Presentations


Edelblut J, et al. Quantifying Preferences for Urea Cycle Disorder Treatments Using a Discrete-Choice Experiment. SIMD March 2023

Steiner R, et al. ACER-001: A Potential Alternative to Sodium and Glycerol Phenylbutyrate for Treatment of Urea Cycle Disorders. GMDI May 2022

Cederbaum S, et al. Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders. GMDI May 2022

Steiner R, et al. The Pharmacokinetics of Taste-Masked Sodium Phenylbutyrate (ACER-001) for the Treatment of Urea Cycle Disorders Under Fasting and Fed Conditions in Healthy Volunteers. SIMD April 2022

Cedarbaum S, et al. Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders. SIMD April 2022

Osaka S, et al. A randomized trial to examine the impact of food on pharmacokinetics of 4-phenylbutyrate in healthy volunteers. Mol Genetics and Metab. Feb. 2021

Nakano S, et al. Effect of food on the pharmacokinetics and therapeutic efficacy of 4-phenylbutyrate in progressive familial intrahepatic cholestasis. Sci Rep 9, 17075 (2019)

Shchelochkov et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver, and provider perspectives


Zubarioglu T, et al. Impact of sodium phenylbutyrate treatment in acute management of maple syrup urine disease attacks: a single-center experience. J Pediatr Endocrinol Metab. 2020 Nov 11;34(1):121-126.

Brunetti-Pierri et al. Phenylbutyrate therapy for maple syrup urine disease. Hum Mol Genet. 2011 February 15; 20(4): 631–640

Strauss KA, et al. Maple Syrup Urine Disease. In: Pagon RA, Adam MP, Ardinger HH, al. e, eds. GeneReviews® [Internet]. https://www.ncbi.nlm.nih.gov/books/NBK1319/: University of Washington, Seattle; 2006. Accessed March 22, 2017

Häberle, J., Boddaert, N., Burlina, A. et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet J Rare Dis 7, 32 (2012)

Chapman, K, et al. (2018). Incidence of maple syrup urine disease, propionic acidemia, and methylmalonic aciduria from newborn screening data. Molecular Genetics and Metabolism Reports. 15. 106-109.

Scaglia F. New insights in nutritional management and amino acid supplementation in urea cycle disorders. Mol Genet Metab. 2010;100 Suppl 1(Suppl 1): S72-6.

L.C. Burrage, et al., Sodium phenylbutyrate decreases plasma branched-chain amino acids in patients with urea cycle disorders, Mol. Genet. Metab. (2014)

ACER-801 (VMS)

Moon, Z. et al. J Factors related to the experience of menopausal symptoms in women prescribed tamoxifen. Journal of Psychosomatic Obstetrics & Gynecology, 2017 Vol. 38, No. 3, 226–235

Prague J., et al. Neurokinin 3 receptor antagonism rapidly improves vasomotor symptoms with sustained duration of action. Menopause. 2018 Aug;25(8):862-869

Challapalli, A, et al. Evaluating the prevalence and predictive factors of vasomotor and psychological symptoms…Clin. and Transl. Radiation Onc. 10 (2018) 29–35

Herman Depypere et al., ENDO2017


Baderkhan, H, et al. Celiprolol Treatment in Patients with vEDS. Euro Journal of Vascular and Endovascular Surgery. Nov. 2020 (Swedish Patient Registry Data)

Poster: Academy of Managed Care Pharmacy Nexus 2019 Tetali et al October 2019

Abstract: Journal of Managed Care & Specialty Pharmacy
Pilot Study to Estimate the Healthcare Cost Associated with Clinical Events in Vascular Ehlers-Danlos Syndrome

Society for Vascular Medicine 2019 Annual Scientific Sessions  Tetali et al  May 2019
Pilot Study to Evaluate Effect of Antihypertensive Therapy on the Rates of Clinical Events in Patients with Vascular Ehlers-Danlos Syndrome

Journal of the American College of Cardiology 2019; 73:194857  Frank Peer Reviewed Publication

Audio summary by Editor-in-Chief of JACC, Dr. Valentin Fuster

Vascular Ehlers-Danlos Syndrome: Publication

Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study


Lancet 2010; 376: 1476-84  Ong  Peer Reviewed Publication

Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial

NORD Poster Presentation  Grabowsky  October 2018

Big Data Analytics to Estimate Prevalence of Ehlers-Danlos Syndrome(Vascular Phenotype) and Rate of Rupture Events

Cardiology in Review 2017; 25: 247-253  Nawarskas  Peer Reviewed Publication

Celiprolol, A Unique Selective Adrenoceptor Modulator

Genetics in Medicine 2014; 16(12): 881-8  Pepin  Peer Reviewed Publication

Survival is affected by mutation type and molecular mechanism in vascular EhlersDanlos syndrome (EDS type IV)

Cardiovascular Research 2019; cvz095  Dubacher  Peer Reviewed Publication

Celiprolol but not losartan improves the biomechanical integrity of the aorta in a mouse model of vascular Ehlers Danlos syndrome

Emetine (Various Viruses)

Sun J, et al. Drug repurposing of pyrimidine analogs as potent antiviral compounds against human enterovirus A71 infection with potential clinical applications. Nature May 2020.

Bojkova D, et al. Proteomics of SARS-CoV-2-infected host cells reveals therapy targets. Nature 2020.

Ianevski A, et al. Antiviral options against SARS-CoV-2 infection. May 2020.

Yang S, et al. Emetine inhibits Zika and Ebola virus infections through two molecular mechanisms: inhibiting viral replication and decreasing viral entry. 

Andersen, P.I., et al. Novel Antiviral Activities of Obatoclax, Emetine, Niclosamide, Brequinar, and Homoharringtonine. Viruses 2019, 11, 964.

Del Puerto et al. Pren. méd. argent., 55: 818, 1968

Annamalai et al. Emetine Hydrochloride in the Treatment of Herpes Zoster. 1968

Shen L, et al. High-Throughput Screening and Identification of Potent Broad-Spectrum Inhibitors of Coronaviruses. J Virol. 2019 May 29;93(12).

Choy et al. Remdesivir, lopinavir, emetine, and homoharringtonine inhibit SARS-CoV-2 replication in vitro. Antiviral Res. 2020 Jun; 178: 104786