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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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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
In February 2023 we were delighted to present a poster at the
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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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WORLDSymposium 2023 poster – Cherry red spot: Evidence that its identification can speed up the diagnosis of GM2 gangliosdosis
In February 2023 we were delighted to present a poster at the
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WORLDSymposium 2021 poster
In February 2021 we were delighted to present our latest poster alongside
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National Organization for Rare Disorders (NORD) Summit 2020 Poster
In October 2020 we were delighted to present alongside our registry partners
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Intrabio IB1001-202 Clinical Trial
The FAQ and clinical trial brochure for the Intrabio IB1001-202 trial can
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NOW Enrolling: GM2 gangliosidosis Gene Therapy Clinical Trial at Queens University
Global enrollment is now open for the TSHA-101 clinical trial at Queens
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