✨Canadian KAND patient, Hendrik, is featured in a YahooLife news article about his journey with KIF1A Associated Neurological Disorder. ❤️Take a look~ https://lnkd.in/guEwBSzE
KIF1A.ORG
Non-profit Organizations
Urgently working to support discovery of treatments for children living with KIF1A Associated Neurological Disorder.
Über uns
KIF1A.org is a nonprofit 501(c)(3) organization working to improve the lives of individuals and families affected by KIF1A Associated Neurological Disorder (KAND) and accelerating research to find treatments and cures. KIF1A Associated Neurological Disorder (KAND) is an ultra-rare neurodegenerative disease caused by variants in the KIF1A gene. Mutations in KIF1A cause a constellation of medical complexities, including cognitive impairment, cerebellar atrophy, ataxia, spastic paraplegia, optic nerve atrophy and epilepsy. KIF1A.ORG works with members of the rare disease community to enable research and discovery of treatments for this generation of people living with KIF1A Associated Neurological Disorder.
- Website
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http://www.KIF1A.org
External link for KIF1A.ORG
- Industrie
- Non-profit Organizations
- Größe des Unternehmens
- 2-10 Mitarbeiter
- Typ
- Nonprofit
- Gegründet
- 2016
- Spezialitäten
- rare disease, fundraising, nonprofit, pediatric medicine, and patient advocacy
Standorte
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Primäre
Wegbeschreibung erhalten
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Wegbeschreibung erhalten
New York, US
Employees at KIF1A.ORG
Aktualisierungen
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KIF1A.ORG reposted this
As she approaches two years of treatment, KIF1A patient Susannah is experiencing fewer seizures, reduced falls, and improved cognition. STAT New's Jonathan Wosen, PhD highlights these outcomes and details similar progress being observed in additional n-Lorem patients. https://lnkd.in/gqbgW3Bu
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✨ Exciting News! ✨ We are thrilled to announce the publication of the **first-ever KAND ASO study**! This groundbreaking research marks a significant milestone in the fight against KAND, and it would not have been possible without the incredible collaboration and dedication of our team and partners. A heartfelt thank you to Dr. Chung and Dr. Bain for leading the charge with unmatched expertise and passion. We also extend our deepest gratitude to n-Lorem for creating and providing the ASO drug that made this trial possible. And most importantly, our utmost respect and appreciation go to the pioneering family and patient who courageously participated in this trial. Your bravery paves the way for future breakthroughs and brings hope to many. Together, we are making history and bringing hope to the KAND community! #KIF1A #KANDResearch #MedicalBreakthrough #Gratitude #HopeForTheFuture #bainbrainlab #nloremfoundation #wchunglab #KANDASO #ASO #nlorem Article: https://lnkd.in/gAYuvNeB
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Awesome. Thank you BIO and John F. Crowley! Go, Sloane, Susannah, and every family! Always relentless in our science, community and advocacy. Global Genes, Chan Zuckerberg Initiative, Columbia Children's Health #CareAboutRare #RareAsOne #Hope #BIO
PATIENT PERSPECTIVES....this week at BIO we hosted an amazing Dad, my friend and NYC Firefighter, Luke Rosen, to talk about his family's journey into the world of rare diseases. In 2016, Luke and his wife Sally faced the diagnosis of their daughter Susannah ("Suzy") with a rare and fatal neurological disease, KIF1A. Back then, she was only the 7th child in the world known to have this devastating disease. There was no hope. Luke, Sally, family and friends were determined to find more children with KIF1A, and to advance science to a treatment- and ultimately a cure. Through their amazing efforts and their not-for-profit KIF1A.ORG, there are now more than 650 kids with a KIF1A diagnosis. Suzy has now received an experimental small molecule therapy that is bringing hope to her and others. This picture of Suzy (in the red shirt) was taken yesterday at Columbia Children's Hospital in NYC. She is greeting a new friend, little Sloane, who also lives with KIF1A. Sloane is there to receive the same experimental treatment for the first time. Amazing man and family who have brought hope to a small but incredibly important part of the world of genetic medicine. A story of love, hope, persistence, science, medicine, faith and biotechnology. New York City Fire Department Global Genes EveryLife Foundation for Rare Diseases Biotechnology Innovation Organization Columbia Children's Health #biotechnology. #science #medicine #faith #hope
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Go, Maddox! You’re going to make a great firefighter. ♥️🚒
Big thanks to our brothers and sisters at the Fullerton Fire Department for making Maddox smile! You guys took time to give a #RareDisease warrior living with #KIF1A an incredible day at the firehouse and on the rigs. Thanks Fullerton Fire for your community impact! Stay safe out there. #FirefightersForPatients
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Thank you The Jackson Laboratory for being #Relentless for the KIF1A community and all of our families! We wouldn’t be where we are today without your world-class expertise, science, collaboration, and tireless work! JAX is family to every rare community. ♥️ #RareAsOne #CareAboutRare
Susannah's family's journey to create a mouse model, and ultimately join a clinical trial, started with The JAX Center for Precision Genetics, a foundational grant for the JAX Rare Disease Translational Center. Learn more at https://lnkd.in/enqgqmX8 KIF1A.ORG #jaxrare #weneedamouse #JAXRDTC
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#WarriorWednesday. Warrior Grace!
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🌈At just 16 months old, Emi Wilkerson lost her precious life to KAND on July 22, 2022. In Emi's honor, her mother, Michelle, has written a children's book featuring her beloved daughter. In the book, Emi helps a little monster named Froggles discover self-love and acceptance while spreading joy and kindness to everyone around her. ✨Currently, Michelle needs our help to launch her book and gift set (which includes a puzzle, and stuffed versions of Emi and Froggles). Her fundraising goal is $10,000 USD, which will allow her to order and sell the book and gift set. In addition, a portion of the proceeds from each sale will fund dreams (like Make A Wish) for sick kiddos, including those in the KAND community. Backer Rewards include: - $25 - Book - $50 - Book and Froggles - $50 - Book and Emi - $100 - Gift Set (Book, Froggles, Emi, Puzzle) - $250 - Book Signing + Gift Set - $500 - Classroom Sponsorship - $1000 - Class Sponsorship + Gift Set 🙏🏻Please help Michelle honor Emi by visiting her Kickstarter campaign and becoming a supporter! Supporters will only be charged if the fundraising goal is achieved by August 15. Let's make this dream a reality! With hope and love, KIF1A.ORG https://lnkd.in/gPKZ-RM5
Froggles: The Cutest Little Monster You Ever Did See by Michelle Wilkerson — Kickstarter
kickstarter.com