A Challenge Met, Million Lives Elevated

Pioneering groundbreaking research, cutting-edge innovation, and unwavering commitment to conquer Rett.

What is Rett Syndrome?

Taming Rett’s Frontier

Rett Syndrome shatters lives; stealing the ability to communicate, hand use, and mobility. It primarily affects young girls, causing them to feel limited in their physical abilities and unable to express the intelligence and emotions they possess.
The pain and frustration of watching a child struggle with this rare genetic neurological condition is heart-wrenching. Your involvement will enhance the research and developmental approaches needed to discover a cure, rekindle hope, and give our children a brighter future they deserve.

Every 1 in 10,000 girls is born with Rett syndrome.
Above 350,000 women suffer from Rett syndrome globally.
Every 2 hours, a child is born with Rett Syndrome.

Our Calling

Leaving Rett Behind

Leading at the forefront of medical innovation, we are a community of dedicated professionals driven by a shared vision to make Rett a thing of the past. Our deep sense of purpose instils a relentless drive to push scientific frontiers by integrating cutting-edge research with the very core of what makes us human - compassion.

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Our Three-Pronged Approach

Hope springs for treating Rett

Our holistic approach to combating Rett Syndrome involves a three strategies designed to enhance the well-being of those impacted. This approach aims to address physical, cognitive, and behavioral challenges to create a comprehensive and effective treatment plan.

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Gene therapy/gene editing

Presents and corrects functional MECP2 gene, thereby restoring normal cellular function and mitigating pathological symptoms.

RNA technologies

Utilises RNA-based modalities to reestablish normal MECP2 gene function and ameliorate pathological symptoms.

Repurposing drugs/small molecules

Identifies existing molecules potentially to alleviate symptoms by modulating the function of the affected pathway.

Our Latest Advancements

Stay informed of the cutting-edge publications and clinical trials conducted by renowned professors and researchers.

Publications

A Novel In Vitro Modeling System for the Study of MeCP2 Pathology and Treatment Response

Unravel the role of non-neuronal cells, i.e. the cells surrounding neurons in the brain, in the pathogenesis of neurodegenerative disorders such as motor neuron disease/ALS, Parkinson’s, Alzheimer’s, but also Rett Syndrome and Friedreich ataxia.

A Novel In Vitro Modeling System for the Study of MeCP2 Pathology and Treatment Response

Unravel the role of non-neuronal cells, i.e. the cells surrounding neurons in the brain, in the pathogenesis of neurodegenerative disorders such as motor neuron disease/ALS, Parkinson’s, Alzheimer’s, but also Rett Syndrome and Friedreich ataxia.

A Novel In Vitro Modeling System for the Study of MeCP2 Pathology and Treatment Response

Unravel the role of non-neuronal cells, i.e. the cells surrounding neurons in the brain, in the pathogenesis of neurodegenerative disorders such as motor neuron disease/ALS, Parkinson’s, Alzheimer’s, but also Rett Syndrome and Friedreich ataxia.

Clinical trials

Turning the tide for Rett syndrome through science and discovery

Leading the fight against Rett Syndrome, clinical trials are paving the way to new treatments.

Taysha gene therapies is testing TSHA-102, a truncated gene therapy in Phase 1 trials.
Neurogene's NGN-401 offers hope with its full-length MECP2 gene therapy.
UPenn and partners are exploring AAV9 & Lentivirus vectors.
Bean therapeutics & Powerlabs use CRISPR-Cas9 technology to make progress.
Activation of the back up MECP-2 copy on the X-chromosome is bringing us ever closer to a cure.

A New Hope for Rett Syndrome

Today’s Challenge, Tomorrow’s
Accomplishment

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