The Voice of the Patient Report from the GM1 Gangliosidosis Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD)
You can now download and read the touching and impactful summary of input from GM1…
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About the GM1 Gangliosidosis Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting with FDA
The GM1 Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD) was held on Friday, October 14 as…
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Rare Disease Day Photo Contest Entry Form
The 2023 Cure GM1 RDD Photo Contest Calling all photographers! Photo submissions for the 2023…
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Cure GM1 Caregiver Preferences Study has been published
Our Caregiver Preferences study, The caregivers’ assessments of symptom impact and most important symptoms to…
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Strahinja’s Story
Hello, I’m Strahinja, 19 months old from Switzerland. I have two older brothers who are fortunately…
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Theodora’s Story
Theodora was born in October 2021. She is a very happy and smiley baby. She…
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Camden’s Story
Camden is a happy child. He loves people. Hearing certain names lights his face up….
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Sweet Iris’ Story
About Sweet Iris Iris was diagnosed with Juvenile GM1 when she was 5 and a…
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Kinley and Kennedy’s Story
Our Journey to Diagnosis By Kylie Harrison “Two of your children have a fatal, untreatable…