Grace Science

@gracescience

We are changing how scientific research is done. By working to find a cure for NGLY1 Deficiency, we’re helping those suffering from countless other diseases.

gracescience.org
Joined June 2014
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  1. Mar 23

    Another big step forward in ! Scientists in the UK have successfully reversed severe vision loss in 2 patients. They used to develop a specially engineered retinal patch. Lots of hope for treating loss in the future:

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  2. Mar 22

    Keep up the great work!

    NGLY1 PerlQuest Stage 3 update from and the nematode team
    3 retweets 10 likes
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  3. Mar 22

    is v. important for patients. Most of them suffer from alacrima & dry eyes that can lead to corneal scarring. Families need good eye drop & gel regimens to keep kids' eyes moist. Like JJ in this pic, some patients also wear glasses to improve . Very cute!

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  4. Mar 21

    Urine oligosaccharide has been discovered as a new & effective way to identify individuals with Deficiency. A big thanks to all the families that provided samples for this project! Read more here:

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    2 retweets 7 likes
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  5. Mar 20

    Starting off with some ! This is Grace riding at the NCEFT, where she has been riding since 23 months old. Her immense in motor skills, leg/core strength & balance continues to stun us. Horse therapy is an incredibly helpful tool for patients.

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  6. Mar 17

    Scientists have created a new gene editing tool called CasRx, targeting RNA instead of DNA. Could help in the development of new & investigation of functions.

    1 retweet 2 likes
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  7. Mar 15

    Our photo series around the world continues- featuring talented & dedicated scientist Dr. Tadashi Suzuki in Japan. Tadashi found & studied NGLY1’s significance 2 decades before any patient was diagnosed & is an incredible asset to our team. Keep up the gr8 work!

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    1 reply 2 retweets 10 likes
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  8. Retweeted
    Mar 14

    Stage 3 (novel compound screening) is in full swing for our NGLY1 Deficiency PerlQuest with ! We're screening NGLY1 mutant worm and fly larvae in the presence of the proteasome inhibitor bortezomib. Here's bortezomib dose response data for png-1 mutant worms

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    1 reply 7 retweets 18 likes
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  9. Mar 14

    Scientists have developed a new method to efficiently generate large numbers of neurons from patients w/ diseases such as Parkinson's Huntington’s & Alzheimer’s. Leads to better disease models to study & screen for new treatments:

    4 retweets 5 likes
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  10. Mar 13

    Recent studies show that the proliferation & improvement of sequencing techniques is leading to great progress in how diseases are diagnosed & treated:

    7 retweets 5 likes
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  11. Mar 12

    Congrats to Dr. Huda Zoghbi for winning the 6th annual Ross Prize in Molecular Medicine! Well deserved. She is 1 of our key advisors & we're excited to see more of her groundbreaking work for neuro & genetic - also helpful for Deficiency.

    1 retweet 8 likes
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  12. Mar 9

    Another vital application of : scientists have used it to help identify genes that play critical parts in the neurodegenerative disease . Allows for the identification of new therapeutic targets.

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  13. Mar 8

    Smile, it's your ! Please join us in wishing Quentin from France a Happy 5th Birthday 🎉🎂🎈We hope you have a wonderful day. Bon anniversaire Quentin!

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  14. Mar 8

    Happy featuring this wonderful group of They nurture our children with love & care everyday & push science forward despite challenges they face. Moms, thank you for ur big hearts, unconditional love & perseverance. Stay strong!

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  15. Retweeted
    Mar 6

    In the same press release, we announced a new PerlQuest for RPS6KA3 and the chair of the Coffin-Lowry Foundation reached out. Instead of companies and researchers recruiting patients, in rare diseases patients recruit companies and researchers.

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    3 replies 10 retweets 31 likes
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  16. Mar 6

    Human pluripotent can self-renew & develop into all major cell types, allowing them to be gr8 & treatment tools. Culturing them in large quantities is quite expensive, but scientists have now developed a more cost-effective culture:

    1 reply 2 likes
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  17. Mar 5

    Researchers found a way to reverse by engineering mice that lose BACE1 enzyme. This prevents the buildup of amyloid plaques in the that kill neurons. Not without drawbacks, but a start to finding a cure for this disease:

    3 retweets 6 likes
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  18. Mar 2

    Interested in learning more about 1 of our key scientists, ? Check out his Q&A here. Hamed & his team are very dedicated to understanding & curing . Grateful to have him on our team:

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  19. Mar 1

    All to start off the new month! Happy from Kristen & Grace. Nothing better than some quality mother-daughter time, while continuing to work hard at the 3 week therapy session & repping our favorite motto: !

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  20. Retweeted
    Feb 28

    So proud to support in its groundbreaking work. "But for a little grace, there may be a cure for her yet."

    33 replies 247 retweets 1,026 likes
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