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Two Families Grapple with Rare Disease Diagnosis, Finding Treatments

By Dana Talesnik
Isabella and Julia Flysjo, age 4, are part of a first-in-human gene therapy trial at the Clinical Center for their rare disease.
Photo: Niclas Flysjo
It’s agonizing for parents to watch their children suffer and not know how to help them. When parents learn their child has a rare disease, consumed with grief and fear, they try to navigate the confusing medical lingo for diseases that have limited resources and few, if any, effective treatments.
Two families were among those who shared their rare disease journeys during NIH’s Rare Disease Day virtual event on Mar. 1. In both cases, their babies were born seemingly healthy. Then, around age 2, worrisome symptoms appeared that led these families to seek a medical evaluation. ....

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Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase ...

Press release content from Globe Newswire. The AP news staff was not involved in its creation.
Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase .
Sio Gene TherapiesDecember 15, 2020 GMT
- Generally well-tolerated with a favorable safety profile in five patients
- Serum beta-galactosidase enzyme activity increased in all patients at all timepoints between Day 7 and Month 6, representing an approximate doubling in enzyme activity after gene transfer
- At Month 6, enzyme activity restored to 23-57% (mean: 38%) of normal reference levels
- All five children demonstrated signs of clinical disease stability as assessed by Vineland-3 Growth Scale Value, Upright and Floor Mobility, and Clinical Global Impression (CGI) scales ....

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