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Economic burden of congenital athymia in the United States for patients receiving supportive care during the first 3 years of life

Congenital athymia is an ultra-rare pediatric condition characterized by the lack of thymus in utero and the naïve T cells critical for infection defense and immune regulation.

ELEVATE 2: A Multicenter Study of Rodatristat Ethyl in Patients with WHO Group 1 Pulmonary Arterial Hypertension

Rodatristat ethyl is a first in class prodrug for rodatristat, a potent inhibitor of tryptophan hydroxylase, the rate limiting enzyme in the biosynthesis of serotonin from dietary tryptophan.

Defining the Clinical, Emotional, Social, and Financial Burden of Congenital Athymia

This cross-sectional study of adult caregivers of patients with congenital athymia used both a quantitative survey and qualitative interviews. Caregivers of patients currently receiving supportive care responded to questions about the past 12 months.

Directed evolution of adeno-associated virus to an infectious respiratory virus

Respiratory viruses evolve to maintain infectivity levels that permit spread yet prevent host and virus extinction, resulting in surprisingly low infection rates. Respiratory viruses harnessed as gene therapy vectors have illustrated this limitation. We used directed evolution in an organotypic human airway model to generate a highly infectious adeno-associated virus.

Impact of Urinary Incontinence Related to Overactive Bladder on Long-Term Care Residents and Facilities: A Perspective From Directors of Nursing

The current study assessed the impact of urinary incontinence (UI) on residents, staff, care processes, and quality measures in long-term care (LTC) settings.

Vibegron for the Treatment of Patients with Dry and Wet Overactive Bladder: A Subgroup Analysis from the EMPOWUR Trial

Overactive bladder (OAB) is characterized by urgency and frequency with (OAB wet) or without (OAB dry) urge urinary incontinence (UUI). In the phase 3 EMPOWUR trial, vibegron—a selective β3-adrenergic receptor agonist for the treatment of OAB—significantly improved daily number of urgency episodes and micturitions vs. placebo (P < 0.01).

An Evaluation of the Efficacy and Safety of Vibegron in the Treatment of Overactive Bladder

Pharmacologic treatment for overactive bladder (OAB), which is characterized by bothersome symptoms such as urgency and urge urinary incontinence (UUI), includes anticholinergics and β3-adrenergic receptor agonists.

Interpretation of the Meaningfulness of Symptom Reduction with Vibegron in Patients with Overactive Bladder: Analyses from EMPOWUR

Reductions in bothersome symptoms of overactive bladder (OAB) demonstrate improvement in clinical trials, but patient perception of meaningfulness of such improvement is lacking.

Effects of vibegron on ambulatory blood pressure in patients with overactive bladder: results from a double-blind, placebo-controlled trial

Patients were randomized to once-daily vibegron 75 mg or placebo for 28 days and underwent ambulatory BP monitoring. The primary endpoint was change from baseline (CFB) to day 28 in mean daytime ambulatory systolic BP (SBP).

A trial design to maximize knowledge of the effects of rodatristat ethyl in the treatment of pulmonary arterial hypertension (ELEVATE 2)

Serotonin plays a key role in the development and maintenance of the pathobiology associated with pulmonary arterial hypertension (PAH). Platelet-driven and locally produced serotonin from lung tissue and arterial endothelial cells induce excessive growth of pulmonary artery smooth muscle cells.

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