Editor's Note: Patient Worthy is honored to share this story, submitted to us by Richard Poulin III. Late one night, a seemingly ordinary Facebook post changed the course of our…
Children all across the globe enrolled in a study to evaluate Upstaza, a gene therapy medication, for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. As part of their…
In November 2022, the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) approved Upstaza (eladocagene exuparvovec) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The product, which…
When Richard Poulin's daughter Rylae-Ann was born, it wasn't long before he and his wife began to wonder if something was wrong. They both felt confident in understanding the…
When Rylae-Ann was one year old, her parents had to hold her in an upright position at night in order for her to breathe. Rylae-Ann had a rare genetic disease,…
The young patients began laughing, and some even began to speak and walk. Imagine the excitement among the hospital staff when they realized that a new medication brought about…
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