Clinical trials

Investigator

Adolfo Ramirez-Zamora

Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties. There are different kinds of SCA and this study will focus on types 1, 2,3, and 6 (SCA 1, SCA 2, SCA 3 , also known as…

Investigator

Sub Subramony

Ages

6 Years - N/A

Sexes

All

This is a natural history study prospectively investigating neuroimaging markers of disease progression in children and adults with Friedreich ataxia (FA). There will be three assessment periods (baseline, 12 and 24 months). The study will include…

Ages

5 Years - N/A

Sexes

All

Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization (WHO)…

Investigator

Ali Ataya

Ages

18 Years - N/A

Sexes

All

160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim or placebo for 48 weeks. Subjects completing the 48 week placebo-controlled period will receive…

Investigator

Ali Ataya

Ages

18 Years - N/A

Sexes

All

The main objective of the study is to assess the effect of treprostinil palmitil inhalation powder (TPIP) compared with placebo on pulmonary vascular resistance.

Investigator

Ali Ataya

Ages

18 Years - 75 Years

Sexes

All

Study ROR-PH-303, ADVANCE EXTENSION, is an open-label extension (OLE) study for participants with WHO Group 1 PAH who have participated in another Phase 2 or Phase 3 study of ralinepag.

Investigator

Ali Ataya

Ages

18 Years - N/A

Sexes

All

This study aims to evaluate the efficacy and safety of inhaled treprostinil in subjects with sarcoidosis-associated interstitial lung disease and pulmonary hypertension.

Investigator

Ali Ataya

Ages

18 Years - 99 Years

Sexes

All