23
Apr
GM2 Gangliosidosis Community Response to the FDA regarding AQNEURSA
You can read our letter to the FDA by clicking on the file.
Read more
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
Read more
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
Read more
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,
Read more
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
Read more
29
May
Study identifies red flags of rare disease
Published in Orphanet Journal of Rare Diseases 21st May 2024: A new study, by Medics4RareDiseases,
Read more
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
Read more
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
Read more
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
Read more