Research’s Promise

Each Breakthrough Brings Us Closer to a Cure*
Stephanie’s Hope specifically funds research through the Friedreich’s Ataxia Research Alliance. All donations generated from the Stephanie’s Hope Holiday Boutique go directly to FARA.

Thanks to the committed efforts of many FA scientists, we now understand the cause of FA and specific mechanisms leading to damage in patients, such as gene mutation, decreased frataxin production, iron sulfur cluster formation, and mitochondrial dysfunction. FARA is supporting the development of treatments aimed at each of these different mechanisms of damage. Because it is based on solid basic science discovery, this targeted approach to treatment has great potential. Furthermore, FA researchers believe that treatment will come in the form of a “cocktail” therapy – meaning that therapies aimed at the different mechanisms of damage have the potential to be used in conjunction with one another to treat the disorder. For further information on the current research areas, visit FARA’s Research Pipeline.

“Just nine years ago when Stephanie’s Hope first started the Research Pipeline only had one clinical trial and three potential treatments/approaches. Now in 2014, we have eight drugs at various stages of clinical trials and sixteen potential treatments/approaches. This progress would not be possible without the support of grassroots fundraising efforts like Stephanie’s Hope. FARA’s ability to fund research, share resources and build partnerships with government agencies, pharmaceutical companies and other organizations is based on support and resources that come from individuals and communities rallied around fighting FA. Through your direct support of Stephanie’s Hope as a volunteer, donor, vendor, buyer and supporter you are directly contributing to this progress.”
-Jennifer Farmer, MS, CGC, Executive Director/FARA

For further information on specific research grants FARA has awarded in the past several years of
Stephanie’s Hope, please visit FARA’s Grants Awarded page.

Research Ripple Effect*

Dr. Harold Varmus, former Director or the National Institutes of Health, gave the following testimony before the House Commerce Subcommitte on Health and Environment in 1998:

“The story of Friedreich’s ataxia illustrates how many areas of clinical and basic research can come together in unexpected ways. In this rare disorder, research involving neuroscience, genetics, clinical medicine, molecular biology, and even biology of yeast and bacteria are converging. The findings offer insights to basic biology and to many other disorders, and illustrate the importance of understanding the mechanism of disease in order to devise treatments.”

Insights into Friedreich’s ataxia could prove beneficial to understandings in other diseases such as:

  • Muscular Dystrophies (ALS, SMA)
  • Parkinson’s Disease
  • Huntington’s Disease
  • Alzheimer’s Disease
  • Fragile X Syndrome
  • Mitochondrial diseases (MELAS, MERF, Lebers)
  • Ataxia-telangiectasia
  • Cerebellar ataxias
  • Diabetes
  • Stroke

The promise of research, along with Stephanie’s Hope and FARA, is to continue to strive towards the goal of finding treatment and a cure for Friedreich’s Ataxia and to bring hope in the meantime.

“The most important thing about research is people stepping up to the plate to participate. There are so many good ideas for studies with new and promising drugs or studies of the mechanism of nerve cell stress in FA (which will lead to more promising drugs), but without people being willing to sacrifice time and energy to join up, those studies will never happen.” -Susan L. Perlman, M.D. Clinical Professor of Neurology/UCLA; Director, Ataxia Center and HD Center of Excellence.

*Information taken directly from