Publications

Primary Hyperoxaluria Type 1 (PH1)

Distinguishing characteristics of pediatric patients with primary hyperoxaluria type 1 in PEDSnet

Journal of Pediatric Urology

Author(s)

Gregory E. Tasian, Kimberley Dickinson, Grace Park, et al

Acute Hepatic Porphyria (AHP)

Clinical features of acute attacks, chronic symptoms, and long-term complications among patients with acute hepatic porphyria in Japan: a real-world claims database study

Orphanet Journal of Rare Diseases

Author(s)

Yutaka Horie, Yuka Yasuoka & Tomohide Adachi

Transthyretin Amyloidosis (ATTR)

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis

New England Journal of Medicine

Author(s)

Mathew Maurer, Parag Kale, Marianna Fontana, et al

Primary Hyperoxaluria Type 1 (PH1)

Qualitative assessment of the patient experience of primary hyperoxaluria type 1: an observational study

BMC Nephrology

Author(s)

David Danese, Diana Goss, Carla Romano, et al

Transthyretin Amyloidosis (ATTR)

Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation

Clinical Pharmacokinetics

Author(s)

Prajakta Badri, Bahru Habtemariam, Megan Melch, et al

Transthyretin Amyloidosis (ATTR)

Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

Neurology and Therapy

Author(s)

Laura Obici, Senda Ajroud-Driss, Kon-Ping Lin, et al

Cardiovascular (CV)

Zilebesiran, an RNA Interference Therapeutic Agent for Hypertension

New England Journal of Medicine

Author(s)

Akshay Desai, David Webb, Jorg Taubel, et al

Acute Hepatic Porphyria (AHP)

Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial

Journal of Hepatology

Author(s)

David J Kuter, Herbert L Bonkovsky, Susana Monroy, et al

Transthyretin Amyloidosis (ATTR)

Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy

Expert Opinion on Pharmacotherapy

Author(s)

Madeline Merkel, David Danese, Chongshu Chen, et al