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.
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
Have you made your #ride4riaan donation yet to sup
Our inspiration Riaan is 32 months-old today! A
As we celebrate our inspiration Riaan turning 32 m
Research update for our Cockayne syndrome drug rep
A rare disease family's trip to the zoo, with many
Our inspiration Riaan listens attentively to his p
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CS caregivers/families: Midwest Genetics Network
Our inspiration Riaan tries his hand at basketball
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In all areas of our work, we are embarking on firs
Summer is here in the Northern Hemisphere! But it'
Our inspiration Riaan enjoys his first close-up fi
"Being the father of a rare human being is the mos
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A very special Happy Father's Day to all of the am
A boy turns 2.5 years-old. Happy 2.5 years to our
Meeting the incredible Kasey Woleben of @therarevi
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