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  • What is GM2?
    • What is Tay-Sachs?
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    • Community respite weekend
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Author: DanielLewi

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Azafaros RAINBOW study update: Completion of 12 week phase 2
12 Mar
DanielLewi0 Comments

Azafaros RAINBOW study update: Completion of 12 week phase 2

Azafaros has just announced the successful completion of its 12-week Phase 2
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New podcast episode: Rare Revelations – Rare Disease Day Edition featuring Patricia Durao
07 Mar
DanielLewi0 Comments

New podcast episode: Rare Revelations – Rare Disease Day Edition featuring Patricia Durao

In the latest episode of the CATS Foundation Rare Revelations podcast, listeners
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Rare Disease Day 2024!
29 Feb
DanielLewi0 Comments

Rare Disease Day 2024!

Today, as we come together to shine a light on rare diseases
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The CATS Foundation at the Externally-Led, Patient-Focused Drug Development (EL-PFDD) Meeting for GM2
22 Feb
DanielLewi0 Comments

The CATS Foundation at the Externally-Led, Patient-Focused Drug Development (EL-PFDD) Meeting for GM2

Last week, the Cure & Action for Tay-Sachs (CATS) Foundation had the incredible opportunity
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The CATS Foundation at WORLDSymposium 2024 – a review of a great week of science and research
19 Feb
DanielLewi0 Comments

The CATS Foundation at WORLDSymposium 2024 – a review of a great week of science and research

We're thrilled to share the highlights from our experience at the recent
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Poster presented at the WORLDSymposium 2024 about our Clinical Toolkit project
13 Feb
DanielLewi0 Comments

Poster presented at the WORLDSymposium 2024 about our Clinical Toolkit project

We were excited to share some of our work at the recent
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Patient-Focused Drug Development (PFDD) meeting with the FDA for GM2 Gangliosidosis 
22 Jan
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Patient-Focused Drug Development (PFDD) meeting with the FDA for GM2 Gangliosidosis 

Our friends at the National Tay-Sachs & Allied Diseases Association have been
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WORLDSymposium 2023 poster – Empowering the GM1 and GM2 community with a co-created education resource about a natural history study
28 Feb
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WORLDSymposium 2023 poster – Empowering the GM1 and GM2 community with a co-created education resource about a natural history study

In February 2023 we were delighted to present a poster at the
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WORLDSymposium 2023 poster – Collaboration between patient advocacy and industry to create a master protocol to investigate the novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosides and Ataxia-telangiectasia
28 Feb
DanielLewi

WORLDSymposium 2023 poster – Collaboration between patient advocacy and industry to create a master protocol to investigate the novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosides and Ataxia-telangiectasia

In February 2023 we were delighted to present a poster at the
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