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Research

 

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.

(source http://www.fara.org.au/fara-funded-research)

Nikolaus Friedreich

Nikolaus Friedreich

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.

 

Research Pipeline

The Friedreich’s Ataxia Treatment Pipeline is a visual tool for communicating the progress of research and development on lead therapeutic candidates.


 

New Zealand Patient Registry

The NZ Neuromuscular Disease Registry helps connect people living with FA to clinical trials and other research opportunities.

If you are interested in registering, please email the curator on nznmdregistry@adhb.govt.nz for more information.

 

FA is a rare condition, so without a patient registry to gather details in one place, finding enough people for a meaningful trial can take years, delaying the testing of potential therapies. Through registering, people become readily identifiable and can be contacted quickly in order to accelerate research opportunities. The NZ Neuromuscular Disease Registry is ethically approved, collects only clinical information that is relevant, and does not share any personal details with researchers or pharmaceutical companies but works to promote the interests of people with FA to the research community and to inform people with FA about relevant research opportunities.

Scientific News

The latest news on Friedreich’s Ataxia compiled by FARA