#34: Hopeful update from Stanford Medicine research
Last month, we met with Professors Jeffrey Goldberg and Joyce Liao of Stanford University. The road to treating autosomal dominant optic atrophy (ADOA) has entered an ambitious new phase. In this extensive meeting, we discussed the important steps being taken at Stanford to combat ADOA. This work, made possible in part by […]
#33: PYC-001, hope on the horizon for the treatment of ADOA
During our ADOA day on Saturday, November 1, 2025, Aishwarya Kundu, Senior Director of Translational Research, shared the latest results from PYC. You can read them in this edition of "The Road to Treatment." A short guide for patients and families. ADOA, or autosomal dominant optic atrophy, is caused by a defect in the OPA1 gene. This makes it […]
#32: New insights into ADOA from Stoke Therapeutics
This time we're sharing a special blog post from Dr. Steve Gross, who is developing a promising new drug for ADOA at Stoke Therapeutics. On Patient Day, November 1, 2025, he shared inspiring and important new insights. The key findings: ADOA patients lose vision over time when they […]
#31: From mitochondrial transplantation to hearing problems
Promising research into ADOA(-plus) and mitochondria. In previous posts on the road to treatment, we primarily highlighted research in the United States. But important research into hereditary optic neuropathies such as ADOA and ADOA+ is also underway in the Netherlands. We spoke with René de Coo, affiliated with the mitochondrial research group led by Prof. Bert Smeets […]
#30: ASHA Therapeutics' Approach to Slowing and Partially Reversing ADOA
New Developments in ADOA Research: ASHA Therapeutics’ Approach to Slowing and Partially Reversing ADOA by Peter Makai In late April 2025, volunteers from the Cure ADOA Foundation and ADOAA (our American sister organization) met with representatives from ASHA Therapeutics. ASHA Therapeutics unveiled promising research on two new drug candidates to […]
#29: A breakthrough in retinal cell therapy
Vision Restoration: A Breakthrough in Retinal Cell Therapy Autosomal Dominant Optic Atrophy (ADOA) is a genetic disorder that primarily affects the optic nerves. The optic nerves are critical for transmitting visual information from the eyes to the brain. The condition often leads to progressive vision loss, beginning in childhood or early adulthood. ADOA is most commonly […]
#28: Interview with Christina Eckmann-Hansen about the impact of ADOA
The Impact of ADOA – Interview with Christina Eckmann-Hansen In October, we spoke with Christina Eckmann-Hansen, an optometrist who did her PhD on ADOA at the University of Copenhagen, supplemented with additional research on the disease. While our primary interest is in possible treatments, we find that the diagnosis and impact of ADOA is not […]
The road to treatment 27
New Developments in Optic Nerve Regeneration – Interview with Petr Baranov In June 2024, we interviewed Dr. Petr Baranov, a researcher at the Schepens Eye Research Institute at Mass Eye and Ear, who is working on the development of cell replacement therapy for glaucoma and optic neuropathies. In ADOA, optic nerve damage is the primary cause of […]
The road to treatment 26
PYC: First patient to receive ADOA medication in October 2024 At the end of September, we had a joint meeting with PYC and the Autosomal Dominant Optic Atrophy Association, our sister organization from the US. PYC has been working for some time on a treatment for classic ADOA, in which the OPA1 mutation causes the disease and there are no other symptoms than the […]
The road to treatment 25
How do clinical trials work at ADOA? Right now, several companies are about to start clinical trials (PYC, Stoke, etc.). These are necessary to demonstrate that their treatment actually works. But what steps must be taken before a drug can be prescribed by your doctor? Before a pharmaceutical company initiates clinical […]
