Coverage of our work & advancements in developing gene therapies to treat neurodegenerative conditions.
In the News
There are significant advancements being made in the field of gene replacement therapy, including for diseases impacting the Central Nervous System.
More than 400 gene therapy trials are currently underway in the United States.
A Brief History of Riaan Research Initiative
Riaan Research Initiative partners with Rarebase to identify drugs that may assist patients with Cockayne Syndrome, a fatal neurodegenerative genetic disorder.
Riaan Research Initiative funds Cockayne Syndrome gene replacement therapy research at UMass Chan Medical School
"The partnership of the Medical School's gene therapy team with the Riaan Research Initiative will lead to a first-in-human AAV gene therapy clinical trial for Cockayne Syndrome as fast as humanly possible. Children like Riaan inspire us to work harder every day to overcome and persist through the challenges in research to give them a change at a better life."
— Miguel Sena-Esteves, PhD, UMass Chan Medical School
Recent progress and considerations for AAV gene therapies targeting the central nervous system
Current Clinical Applications of In Vivo Gene Therapy with AAVs
IN THE NEWS
My Son Was Born With An Incredibly Rare Disease & Fighting for His Life Isn’t Anything Like I Imagined
Personal Reflections on Riaan & Riaan Research Initiative: Listen Here
We have already achieved the impossible': fighting a rare childhood disease with hope.
Interview with RRI Founder, Jo Kaur
Scientists Partially Restored a Blind Man’s Sight With New Gene Therapy
An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future
First Gene Therapy for Tay-Sachs Disease Successfully Given to Two Children
Central Utah toddler makes remarkable progress after groundbreaking gene replacement therapy
Our inspiration Riaan just returned from his Make-
Today is Rare Disease Day 2024. We recognize and h
We've all been there. While individually many rare
February is Rare Disease Month! This month, people
“Riaan, a bright, energetic child who loves play
Our work and our inspiration Riaan were recently f
We are heartbroken and devastated by the passing o
We enter this year fully engaged in the battle. Th
As we approach the end of 2023, we feel the urgenc
What if after the worst news, there was actual hop
For many families living with a Cockayne syndrome
End of Year Fundraising! Hope. Treatments. The Im
As we count down the final days of December and th
Check out our latest post on riaanresearch.substac
Happy 4th Birthday to our amazing inspiration Riaa
Last month, we raised a total of $51,649.48 with y
A special thanks from our inspiration Riaan to eve
Thank you to everyone who has supported our work!
"You are not just a drop in the ocean. You are the
Tis the season to give! It's Giving Tuesday! Cock
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© 2024 Riaan Research Initiative is a 501(c)(3) non-profit organization.