Although German physician Nikolaus Friedreich first recognized Friedreich’s ataxia in 1863, it was not until 1996 that two scientific teams discovered the underlying cause of the disease: a mutation in the gene for frataxin protein.
As scientists learn more and more about Friedreich’s ataxia, they are able to direct their research efforts to creating treatments that show the greatest possible potential for treating Friedreich’s ataxia. Research into FA is occurring throughout the world. Many New Zealanders with FA have participated in the Friedreich Ataxia Clinical Research Program led by Professor Martin Delatycki at the Murdoch Children’s Research Institute and the Friedreich Ataxia Clinic at Monash Medical Centre in Melbourne.
Studies include evaluation and analysis of neurological and functional MRI measures, cardiac function, gait, speech, vision and ocular motility, hearing acuity, quality of life and sexual function in individuals with Friedreich ataxia.
This comprehensive clinical research program is one of the sites of the Collaborative Clinical Research Network in Friedreich’s Ataxia (CCRN in FA), which is an international network of clinical research groups working together to advance treatments and clinical care. The network collaborates with pharmaceutical companies, government agencies, other research groups and the patient community to facilitate clinical research and trials needed to identify new therapies.
Partnership with Australia
Due to the small population of New Zealand faraNZ works closely with our larger Australian cousins, fara. The Australian organization helps faraNZ with our research goals and opportunities, and the way we operate for New Zealanders with FA.
The Friedreich’s Ataxia Treatment Pipeline is a visual tool for communicating the progress of research and development on lead therapeutic candidates.
Global Patient Registry
This registry currently holds the demographic and clinical information on more than 3,400 Friedreich’s ataxia patients from across the United States and internationally. This registry was created to serve the patient, physician, and research communities.
The latest news on Friedreich’s Ataxia compiled by FARA