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Inherited Retinal Degeneration

NAC Attack

Posted on December 20, 2023

NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double-Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients with Retinitis Pigmentosa.

Posted in Actively Recruiting, Clinical Trials, Inherited Retinal Degeneration, Kimberly Stepien, Retinitis Pigmentosa

UNI-RARE

Posted on December 20, 2023

Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated with Rare Disease-Causing Genetic Variants.

Posted in Actively Recruiting, Clinical Trials, Inherited Retinal Degeneration, Kimberly Stepien, Retinal Dystrophy

XOLARIS

Posted on December 1, 2020

To gain a better understanding of disease progression over time in male subjects with X-linked retinitis pigmentosa (XLRP).

Posted in Clinical Trials, Inherited Retinal Degeneration, Kimberly Stepien, Past

CHM SURVEY TRIAL

Posted on December 1, 2020

Choroideremia (CHM) Health Outcomes

Posted in Choroideremia, Clinical Trials, Inherited Retinal Degeneration, Kimberly Stepien, Past

Pro-EYS

Posted on September 30, 2020

Rate of Progression in EYS Related Retinal Degeneration (Pro-EYS).

Posted in Clinical Trials, Genetic mutation, Inherited Retinal Degeneration, Kimberly Stepien, Ongoing

AGTC A3

Posted on September 30, 2020

A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of AGTC 402, a Recombinant Adeno-associated Virus Vector Expressing CNGA3, in Patients with Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene

Posted in Clinical Trials, Congenital Achromatopsia, Genetic mutation, Inherited Retinal Degeneration, Kimberly Stepien, Past

AGTC B3

Posted on September 30, 2020

A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients with Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene

Posted in Clinical Trials, Congenital Achromatopsia, Genetic mutation, Inherited Retinal Degeneration, Kimberly Stepien, Past

NIGHT

Posted on September 30, 2020

Natural History of the Progression of Choroideremia Study (NIGHT)

Posted in Choroideremia, Clinical Trials, Inherited Retinal Degeneration, Kimberly Stepien, Past

STAR

Posted on September 30, 2020

A Randomized, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1) (STAR)

Posted in Choroideremia, Clinical Trials, Inherited Retinal Degeneration, Kimberly Stepien, Past
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