Ahead of Rare Disease Day, Illumina is recognizing four rare disease patient organizations around the world. SLC6A1 Connect is the first in our series this month. When Amber Freed was expecting twins ...
You have reached your maximum number of saved items. Remove items from your saved list to add more. Watching videos of their daughter’s birthday, David and Nadine Lipworth can’t recognise the girl ...
TSHA-103 joins portfolio of rare pediatric disease and orphan drug designated product candidates including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome, TSHA-104 for SURF1-associated ...
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8-year-old has disease so rare it doesn’t have a name. Now he’s first in the world to receive treatment (exclusive)
Amber Freed's son Maxwell made history in September when he received treatment for SLC6A1 thanks to her tireless work and a team of dedicated scientists Amber Freed's son Maxwell was diagnosed with ...
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