CATS Foundation https://cats-foundation.org/ Cure and Action for Tay-Sachs Thu, 24 Apr 2025 11:04:01 +0000 en-US hourly 1 https://wordpress.org/?v=6.9.4 43644941 GM2 Gangliosidosis Community Response to the FDA regarding AQNEURSA https://cats-foundation.org/uncategorized/gm2-gangliosidosis-community-response-to-the-fda-regarding-aqneursa/?utm_source=rss&utm_medium=rss&utm_campaign=gm2-gangliosidosis-community-response-to-the-fda-regarding-aqneursa https://cats-foundation.org/uncategorized/gm2-gangliosidosis-community-response-to-the-fda-regarding-aqneursa/#respond Wed, 23 Apr 2025 09:58:04 +0000 https://cats-foundation.org/?p=10825 You can read our letter to the FDA by clicking on the file.

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]]> https://cats-foundation.org/uncategorized/gm2-gangliosidosis-community-response-to-the-fda-regarding-aqneursa/feed/ 0 10825 The CATS Foundation’s “Spot the Stop” Campaign Wins Bronze at PM Society Digital Awards https://cats-foundation.org/uncategorized/the-cats-foundations-spot-the-stop-campaign-wins-bronze-at-pm-society-digital-awards/?utm_source=rss&utm_medium=rss&utm_campaign=the-cats-foundations-spot-the-stop-campaign-wins-bronze-at-pm-society-digital-awards https://cats-foundation.org/uncategorized/the-cats-foundations-spot-the-stop-campaign-wins-bronze-at-pm-society-digital-awards/#respond Mon, 16 Sep 2024 09:02:46 +0000 https://cats-foundation.org/?p=10776 We’re incredibly proud to announce that our Spot the Stop campaign, developed in collaboration with Verve and Four Health, has been recognized with a Bronze Award at the prestigious PM Society Digital […]

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]]> https://cats-foundation.org/uncategorized/the-cats-foundations-spot-the-stop-campaign-wins-bronze-at-pm-society-digital-awards/feed/ 0 10776 Azafaros press release:  Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C https://cats-foundation.org/uncategorized/azafaros-press-release-azafaros-announces-positive-topline-phase-2-study-data-with-nizubaglustat-in-gm2-gangliosidosis-and-niemann-pick-disease-type-c/?utm_source=rss&utm_medium=rss&utm_campaign=azafaros-press-release-azafaros-announces-positive-topline-phase-2-study-data-with-nizubaglustat-in-gm2-gangliosidosis-and-niemann-pick-disease-type-c https://cats-foundation.org/uncategorized/azafaros-press-release-azafaros-announces-positive-topline-phase-2-study-data-with-nizubaglustat-in-gm2-gangliosidosis-and-niemann-pick-disease-type-c/#respond Tue, 16 Jul 2024 16:36:34 +0000 https://cats-foundation.org/?p=10772 We are pleased to share the latest press release from Azafaros related to their study with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C.   The full press release […]

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]]> https://cats-foundation.org/uncategorized/azafaros-press-release-azafaros-announces-positive-topline-phase-2-study-data-with-nizubaglustat-in-gm2-gangliosidosis-and-niemann-pick-disease-type-c/feed/ 0 10772 New podcast episode: Rare Revelations – of a IMD Consultant https://cats-foundation.org/uncategorized/new-podcast-episode-rare-revelations-of-a-imd-consultant/?utm_source=rss&utm_medium=rss&utm_campaign=new-podcast-episode-rare-revelations-of-a-imd-consultant https://cats-foundation.org/uncategorized/new-podcast-episode-rare-revelations-of-a-imd-consultant/#respond Mon, 15 Jul 2024 15:51:48 +0000 https://cats-foundation.org/?p=10762 The latest edition of the Rare Revelations podcast featured Dr María Jose De Castro Lopez, a consultant in Paediatric Inherited Metabolic Diseases at Willink Biochemical Genetics Unit in the UK. […]

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]]> https://cats-foundation.org/uncategorized/new-podcast-episode-rare-revelations-of-a-imd-consultant/feed/ 0 10762 Third Sector Award Nomination – Communications Campaign Of The Year https://cats-foundation.org/uncategorized/third-sector-award-nomination-communications-campaign-of-the-year/?utm_source=rss&utm_medium=rss&utm_campaign=third-sector-award-nomination-communications-campaign-of-the-year https://cats-foundation.org/uncategorized/third-sector-award-nomination-communications-campaign-of-the-year/#respond Fri, 12 Jul 2024 16:12:17 +0000 https://cats-foundation.org/?p=10766 We are very very very happy to share that our “Spot the Stop” campaign developed by the fantastic teams at Verve and Four Health has been shortlisted in the “Communications […]

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]]> https://cats-foundation.org/uncategorized/third-sector-award-nomination-communications-campaign-of-the-year/feed/ 0 10766 Study identifies red flags of rare disease https://cats-foundation.org/blogs/study-identifies-red-flags-of-rare-disease/?utm_source=rss&utm_medium=rss&utm_campaign=study-identifies-red-flags-of-rare-disease https://cats-foundation.org/blogs/study-identifies-red-flags-of-rare-disease/#respond Wed, 29 May 2024 13:33:00 +0000 https://cats-foundation.org/?p=10758 Published in Orphanet Journal of Rare Diseases 21st May 2024: A new study, by Medics4RareDiseases, has identified seven ‘red flags, or clinical clues, that point to a patient having an […]

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]]> https://cats-foundation.org/blogs/study-identifies-red-flags-of-rare-disease/feed/ 0 10758 New podcast episode: Rare Revelations – of Late Onset Sandhoff Disease https://cats-foundation.org/uncategorized/new-podcast-episode-rare-revelations-of-late-onset-sandhoff-disease/?utm_source=rss&utm_medium=rss&utm_campaign=new-podcast-episode-rare-revelations-of-late-onset-sandhoff-disease https://cats-foundation.org/uncategorized/new-podcast-episode-rare-revelations-of-late-onset-sandhoff-disease/#respond Thu, 23 May 2024 17:30:17 +0000 https://cats-foundation.org/?p=10752 The latest edition of the Rare Revelations podcast featured Josh who talked through his diagnosis of Late Onset Sandhoff disease. He told his story about how he was initially misdiagnosed, […]

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]]> https://cats-foundation.org/uncategorized/new-podcast-episode-rare-revelations-of-late-onset-sandhoff-disease/feed/ 0 10752 Sanofi Press Release: Sanofi’s AMETHIST trial for GM2 gangliosidosis discontinued https://cats-foundation.org/uncategorized/sanofi-press-release-sanofis-amethist-trial-for-gm2-gangliosidosis-discontinued/?utm_source=rss&utm_medium=rss&utm_campaign=sanofi-press-release-sanofis-amethist-trial-for-gm2-gangliosidosis-discontinued https://cats-foundation.org/uncategorized/sanofi-press-release-sanofis-amethist-trial-for-gm2-gangliosidosis-discontinued/#respond Thu, 25 Apr 2024 18:01:22 +0000 https://cats-foundation.org/?p=10709 Today it was announced by Sanofi that the AMETHIST phase 3 study of venglustat for the treatment of GM2 gangliosidosis was discontinued based on the absence of positive trends on clinical endpoints. […]

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]]> https://cats-foundation.org/uncategorized/sanofi-press-release-sanofis-amethist-trial-for-gm2-gangliosidosis-discontinued/feed/ 0 10709 “Spot the Stop” for Tay-Sachs and Sandhoff campaign results https://cats-foundation.org/blogs/spot-the-stop-for-tay-sachs-and-sandhoff-campaign-results/?utm_source=rss&utm_medium=rss&utm_campaign=spot-the-stop-for-tay-sachs-and-sandhoff-campaign-results https://cats-foundation.org/blogs/spot-the-stop-for-tay-sachs-and-sandhoff-campaign-results/#respond Mon, 08 Apr 2024 13:31:15 +0000 https://cats-foundation.org/?p=10703 The Campaign: A Resounding Success In the world of advocacy and awareness campaigns, success is often measured not just in numbers, but in impact. One such campaign that exemplifies this […]

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]]> https://cats-foundation.org/blogs/spot-the-stop-for-tay-sachs-and-sandhoff-campaign-results/feed/ 0 10703 Azafaros RAINBOW study update: Completion of 12 week phase 2 https://cats-foundation.org/research-2/azafaros-rainbow-study-update-completion-of-12-week-phase-2/?utm_source=rss&utm_medium=rss&utm_campaign=azafaros-rainbow-study-update-completion-of-12-week-phase-2 https://cats-foundation.org/research-2/azafaros-rainbow-study-update-completion-of-12-week-phase-2/#respond Tue, 12 Mar 2024 16:19:47 +0000 https://cats-foundation.org/?p=10673 Azafaros has just announced the successful completion of its 12-week Phase 2 clinical study, RAINBOW, evaluating their lead asset, nizubaglustat, in patients with rare diseases. This study, conducted in Brazil, […]

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]]> https://cats-foundation.org/research-2/azafaros-rainbow-study-update-completion-of-12-week-phase-2/feed/ 0 10673