BOB体育

Investigator

Adolfo Ramirez-Zamora

Investigator

Carmen Leon Astudillo

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鈥�

Investigators

Matthew R Burns, Sub Subramony

Ages

6 Years - N/A

Sexes

All

To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).

Investigator

Sub Subramony

Ages

18 Years - N/A

Sexes

All

The purpose of this research study is to determine a way to measure frataxin messenger RNA (mRNA) in fluids such as blood and cerebrospinal fluid (CSF) from patients with Friedreich's ataxia (FRDA). The gene mutation in FRDA leads to low levels of鈥�

Investigator

Sub Subramony

Ages

18 Years - 65 Years

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鈥�

Ages

5 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

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鈥�

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