GLOBAL STARGARDT’S DISEASE DAY

Stargardt’s syndrome, also known as juvenile macular degeneration, is a rare genetic disorder that usually develops in young adults and causes a progressive degeneration of the macula, the central region of the retina responsible for vsharp, central and color vision. Consequently, the main symptom of Stargardt’s disease is the loss of central vision and color […]

Novel dual-vector delivery system for Stargardt disease gene therapy

The American biotechnology company Applied Genetic Technologies Corporation (AGTC), specialized in the development of gene therapies for rare diseases, has just announced Stargardt disease as its new preclinical gene therapy program in ophthalmology. Stargardt disease characterized by a progressive loss of central vision affects one in 8,000-10,000 individuals and is the most common form of inherited macular degeneration. […]

Macular Week: Recent advances in pharmacotherapy for Stargardt disease

Macular Week is all about sensitizing and informing patients, associations, professionals and society of latest achievements on macular degenerative pathologies. In this publication, we will focus on the news on orphan drugs that are currently in the clinical or preclinical phase for the treatment of Stargardt disease. Stargardt disease, the most common inherited macular dystrophy, […]

Interview with Margaret Creus – Rare Disease Day

We are celebrating the Rare Disease Day giving voice to our patients and their families. Now is the turn of Margaret Creus, leader of PedRetina, pediatrician and mother of a patient of Stargardt’s disease. Margaret is also a tireless fighter, a commited seeker of innovative solutions and experienced organizer of sports and cultural activities, gathering […]

Interview with Marcela Ciccioli – Rare Disease Day

We believe it is essential to keep a close relationship with all of our patients and know their stories. This is why to celebrate the Rare Disease Day, we have interviewed Marcela Ciccioli, president of Stargardt APNES (Asociación de Pacientes y Padres de Niños con Enfermedad de Stargardt de Argentina) and mother of Victoria, a […]