Publications and Presentations

ACER-001 (UCD/MSUD)

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 et al. Impact of sodium phenylbutyrate treatment in acute management of maple syrup urine disease attacks: a single-center experience (2020).

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

EDSIVO™ (vEDS)

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

Editorial

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

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

Osanetant (iVMS)

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

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

Herman Depypere et al., ENDO2017

Emetine (COVID-19)

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