The road to treatment 24
Taking stock of Idebenone By Peter Makai PhD and Christina Barckhausen PhD Idebenone is a licensed drug for the disease Leber hereditary optic neuropathy (LHON), which is similar to ADOA. Idebenone is therefore one of the oldest and demonstrably safe medications to slow the progression of ADOA. ADOA patients receive this drug in some […]
The road to treatment 23
The Road to Treatment 23 Interview with Tom Schwarz of Harvard We connected with Thomas Schwarz, who is Professor of Neurobiology at Harvard Medical School and Professor of Neurology at the FM Kirby Neurobiology Center, Boston Children's Hospital. Thomas Schwarz and his laboratory are involved in ADOA research and are developing not one, but two […]
The road to treatment 22
The road to treatment 22 At the end of February we had a consultation with Phosphoenix, a Dutch company that is developing a brain implant to restore sight to blind people. The implant relays information from a camera linked to glasses through electrodes that connect to […]
The road to treatment 21
Dr. Astrid Plomp was present on the Anniversary Day on November 18, 2023 and spoke about clinical genetics. What does a clinical geneticist do? The first presentation was on clinical genetics and was given by Dr. Astrid Plomp. Clinical genetics is a medical specialty and field of research concerned with the application of genetic research […]
The road to treatment 20
The road to treatment 20 Fatigue – Ruth van Nispen and Manon Veldman Ruth van Nispen and Manon Veldman were present at the ADOA day and spoke about the EnergEYEZe research, among other things. Fatigue is an important topic for visually impaired people. There are two types of fatigue: acute (temporary) and chronic (long-lasting). Fatigue can be physical, […]
The road to treatment 19
The road to treatment 19 ADOA and ADOA+ On our Anniversary day on November 18 in Doorn, René de Coo of the Nemo Mitochondrial Medicine group, Toxicogenomics department of Maastricht University, explained the operation of ADOA+ and the road to treatment. ADOA is a mitochondrial disease in which the energy factories in the […]
The road to treatment 18
The Road to Treatment 18 PYC at the Gate of Human Studies We recently met with PYC, a company working on a cure for ADOA. In the next six months, PYC will apply for approval to initiate clinical trials with human participants. To grant this consent […]
The road to treatment 17
The road to treatment 17 On our 5th anniversary, Guy Lenaers gave us a presentation about the work he and his colleagues do at the University of Angers (France). The team of Dr. Guy Lenaers, consisting of 60 experts (30 medical professionals and 30 researchers), specializes in hereditary mitochondrial diseases and has unique […]
The road to treatment 16
The Road to Treatment 16 In October we had the pleasure of speaking with Neurophth Therapeutics, a Chinese company developing a gene therapy for ADOA. Recently, Australia's Therapeutic Goods Administration (TGA) approved this program as a drug candidate for clinical investigation, which is necessary to test the drug in ADOA patients. A […]
The road to treatment 15
The Road to Treatment 15 Professor Marcela Votruba of Cardiff University in the United Kingdom, a researcher who discovered the link between ADOA and the damaged OPA1 gene, has spent years researching ADOA. Her research focuses on understanding the disease mechanism of autosomal dominant optic atrophy and designing therapies. […]
