Our guest this week is Paul Nioi, Senior Vice President of Research at Alnylam and we talked about the evolution of RNAi. The post How RNAi is expanding from a therapy of choice for rare disease into treating common conditions appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that ful...| Labiotech.eu
Vicki McCarrell had always dreamed of becoming a mother. When she gave birth at age thirty-eight to her son Sean, life seemed complete. Yes, he had the full facial paralysis typical of Moebius syndrome, but otherwise, he looked perfect to her. Indeed, at the hospital near her home in Van Nuys, California, she was given a diagnosis and not much else. Nobody told her how to feed a baby who could not suck or even warned her that it would be an issue.| Susannah Fox
Jill Dopf Viles suspected a killer was hiding in her family’s genetic code. Symptoms popped up among her siblings, her father, her uncle, her grandmother, but, despite 15 annual visits to the Mayo Clinic, no clinician suggested anything more specific than muscular dystrophy.| Susannah Fox
With time not on their side, CHOC care team pulls out all stops to save life of Emery Kline.| CHOC Pediatrica
Carter and his family started changing the narrative about disability even before he was diagnosed with this rare condition.| Boston Children's Answers
My phone started blowing up with texts from friends and family members yesterday morning, alerting me to a New York Times magazine cover story by Amanda Hess, “My Son Has a Rare Syndrome. So I Turned to the Internet” (gift link). Hess writes beautifully about her experience being dropped into the medical maze and her […]| Susannah Fox
Asa once wondered if he’d ever feel better, experiencing abdominal pain and severe diarrhea since birth. Genomic sequencing changed his life.| Boston Children's Answers
Is myasthenia gravis actually a rare disease? Columnist Shawna Barnes addresses this common question in the MG community.| Myasthenia Gravis News
The post Marvel Biosciences to File for Orphan Drug Designation with the U.S. FDA for MB-204 as a Treatment for Rett Syndrome appeared first on Marvel Biosciences.| Marvel Biosciences
Written by Drs. Phil Hieter and Hugo J. Bellen It is estimated that there are more than 10,000 to 12,000 rare diseases affecting more than 20 million people in the […]| Welcome | Michael Smith Laboratories
In light of Rare Disease Day 2022, columnist Jacqueline Babiarz, who has a daughter with Rett syndrome, considers what it means to be rare.| Rett Syndrome News
KLS medical student and KLS researcher, Michael Hamper, shares his thoughts on what KLS is, how KLS is diagnosed and how KLS is treated.| KLS Foundation