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Participation in a phase 1 clinical trial

Posted By Enny

"Participation in a phase 1 clinical trial"

About: Save Sight Institute Sydney / Sydney Eye Hospital

(as the patient),

About 25 years ago, I first heard that gene therapy was being tested as a potential treatment for genetic eye diseases. Since then, I have held onto the hope that the specific genetic defect affecting me and many members of my family might one day be treatable.

Finding out that clinical trials have now begun for individuals with a defective OPA1 gene is, in my opinion, truly fantastic. Because a genetic disease is passed down to future generations, the prospect of a treatment provides a profound sense of hope for the future.

I feel such gratitude when I think back on my visits to the clinic. I am so glad that such highly skilled professionals have taken on this task. Emily and Chanel stayed in touch every step of the way, ensuring I was always well-informed and had everything I needed.

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Responses

Response from Pauline Rumma, Director, Clinical Services, Sydney and Sydney Eye Hospital last month
Pauline Rumma
Director, Clinical Services,
Sydney and Sydney Eye Hospital

Submitted on 14/05/2026 at 3:32 PM
Published on Care Opinion Australia at 3:36 PM

Dear Enny

Thank you for sharing your recent experience being involved in a Clinical Trial conducted at Sydney/Sydney Eye Hospital.

We are pleased to hear about your positive experience while participating in the PYC genetic trial for autosomal dominant optic atrophy, conducted by Save Sight Institute and Sydney Eye Hospital. Patients participating in our research studies and clinical trials assist us in finding better, more effective treatments. We are pleased that you and your family could be part of this effort.

Clinical trials are a collaborative effort involving clinicians, nurses, researchers, and allied health professionals, all working together to support patient care and the innovation of treatment options.

We are lucky to have such a great team advocating and continuing to work to improve outcomes of patients with these rare genetic conditions.

We will ensure your feedback is shared with the Save Sight Institute research team, and the relevant staff involved in your care.

Dr Pauline Rumma
Director of Clinical Services

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