Open‐label pilot study of interferon gamma‐1b in Friedreich ataxia

@article{Seyer2015OpenlabelPS,
  title={Open‐label pilot study of interferon gamma‐1b in Friedreich ataxia},
  author={Lauren A. Seyer and Nathaniel R Greeley and Dominik Foerster and Cassandra J Strawser and Sarah Gelbard and Yi Na Dong and Kimberly A. Schadt and Maria Grazia Cotticelli and Alicia F D Brocht and Jennifer M. Farmer and R B Wilson and David R. Lynch},
  journal={Acta Neurologica Scandinavica},
  year={2015},
  volume={132},
  pages={15 - 7}
}
This is an open‐label trial of the safety of interferon gamma‐1b (IFN‐γ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia. 

Randomized, double‐blind, placebo‐controlled study of interferon‐γ 1b in Friedreich Ataxia

Evaluated the efficacy and safety of IFN‐γ 1b in the treatment of Friedreich Ataxia through a double‐blind, multicenter, placebo‐controlled trial.

IFN-γ for Friedreich ataxia: present evidence.

Ongoing Phase II and III trials in both adults and children with FRDA showed that IFN-γ-1b was reasonably well-tolerated and improved overall neurological function as measured by the Friedreich Ataxia Rating Scale after 12 weeks of treatment, though the primary outcome measure of frataxin level showed no improvement.

Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia

The dose‐ranging portion of this Phase 2 study assessed the safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia patients.

Safety and Efficacy Of Interferon γ in Friedreich's Ataxia

The treatment was safe, with only 2 serious adverse effects, both spontaneously resolved, and 1 termination as a result of a refusal by the patient to continue the treatment despite resolution of the event, otherwise the treatment was reasonably well tolerated with the occurrence of common adverse events known to be associated with IFNγ treatment.

GIFT-1, a phase IIa clinical trial to test the safety and efficacy of IFNγ administration in FRDA patients

A phase IIa clinical trial, the first in Italy, aimed at assessing both safety and tolerability of IFNγ in Friedreich’s patients and ability to increase frataxin levels in peripheral blood mononuclear cells, found IFnγ was generally well tolerated and the main adverse event was hyperthermia/fever.

Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich's Ataxia: Retrospective Study.

The clinical features, tolerability, and the prognosis of individuals with FRDA to whom IFN-γ was administered in a university hospital were evaluated retrospectively and the results were discussed.

An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich’s ataxia

An open‐label, pilot study of recombinant human granulocyte-colony stimulating factor administration in seven people with Friedreich’s ataxia shows potential for G-CSF therapy to have a clinical impact in people with FA.

Progress in the treatment of Friedreich ataxia.

Emerging therapies in Friedreich's ataxia.

Emerging therapies and future perspectives are discussed, including the need for more precise measures for detecting changes in neurologic symptoms as well as a disease-modifying agent.

Etravirine in Friedreich's ataxia: Lessons from HIV?

The reconstitution of aconitase shows that the etravirine-induced increase in frataxin leads to functional enzymatic improvements that may be disease related, and there are several necessary steps before this medication could be tried widely in patients.

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