Happy Friday!
While the weather this week has been tumultuous, treatment news offers hope to the rare community. This week, we have an announcement about a Scleroderma registry, and PKU treatment news patients can participate in. We also have some updates on Hunter syndrome gene therapy and an early stage of Huntington’s.
Sit back and enjoy this week’s Editor’s Choice.
[one_half]
[/one_half] [one_half_last]
A Scleroderma Registry is Being Launched
Research centers are collaborating with the SRF to create the very first scleroderma patient registry of it’s kind!
Check it out here.
[/one_half_last] [one_half]
[/one_half] [one_half_last]
Data Looks Good For Synlogic’s Experimental Phenylketonuria Drug, And If You’re a Patient, You Can Get Involved
If you’re waiting for a new PKU medication, you might want to check this out.
Read about it here.
[/one_half_last] [one_half]
[/one_half] [one_half_last]
Preliminary Results of Gene Editing Experiment Suggest Potential for Treating Hunter Syndrome
Hunter syndrome is a difficult diagnosis and many families are waiting for hope. New results from a gene therapy study are looking good right now.
Read more here.
[/one_half_last] [one_half] [
[/one_half] [one_half_last]
A Study of Huntington’s Disease in Mice Suggests an ‘Early Stage’ of the Condition
How early should a person with Huntington’s expect to see symptoms? The answer might not be what we previously thought.
Check out the story here.
[/one_half_last] [one_half]We hope you enjoy this week’s Editor’s Choice posts! Don’t forget to share!
Do you have a rare disease experience of your own? Share with us here.
