16
Sep
The CATS Foundation’s “Spot the Stop” Campaign Wins Bronze at PM Society Digital Awards
We’re incredibly proud to announce that our Spot the Stop campaign, developed in collaboration with Verve and
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16
Jul
Azafaros press release: Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C
We are pleased to share the latest press release from Azafaros related to their study
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15
Jul
New podcast episode: Rare Revelations – of a IMD Consultant
The latest edition of the Rare Revelations podcast featured Dr María Jose De Castro Lopez,
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12
Jul
Third Sector Award Nomination – Communications Campaign Of The Year
We are very very very happy to share that our “Spot the Stop” campaign developed
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29
May
Study identifies red flags of rare disease
Published in Orphanet Journal of Rare Diseases 21st May 2024: A new study, by Medics4RareDiseases,
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23
May
New podcast episode: Rare Revelations – of Late Onset Sandhoff Disease
The latest edition of the Rare Revelations podcast featured Josh who talked through his diagnosis
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25
Apr
Sanofi Press Release: Sanofi’s AMETHIST trial for GM2 gangliosidosis discontinued
Today it was announced by Sanofi that the AMETHIST phase 3 study of venglustat for the treatment
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08
Apr
“Spot the Stop” for Tay-Sachs and Sandhoff campaign results
The Campaign: A Resounding Success In the world of advocacy and awareness campaigns, success is
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12
Mar
Azafaros RAINBOW study update: Completion of 12 week phase 2
Azafaros has just announced the successful completion of its 12-week Phase 2 clinical study, RAINBOW,
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