Updates
Notre Dame Day 2020
Psalms 91 Equipped
Southport Mum renews appeal for daughter with rare disorder
Brandon and I found out on June 20th, 2017 that we were expecting our first baby. The entire pregnancy was “textbook,” in fact, my doctor always called it “boring,” and we were grateful for that. With every passing week my “What to Expect” app kept…
MESA — Guided by faith, an Arizona family is on a mission to raise awareness of and eradicate a rare genetic condition that impacts about 500 children every year, including their toddler son. Along the way, they also hope to bring comfort and support to…
The National Institutes of Health has once again renewed a five-year contract with the University of Pennsylvania’s gene therapy program, which will receive $13 million to support the advancement of gene-therapy research to the clinic. The contract was awarded by National Heart, Lung and Blood…
Twenty-four hours after the birth of their son, a Mesa couple’s lives were turned upside down after learning he was born with a rare medical condition. For weeks, in between hospital stays and new challenges, Brandon and Chancee Culp never gave up hope and now…
A group of 20 individuals from Kew McDonalds in Southport have volunteered to complete a sponsored Snowdon climb on the 30th of October 2019. We are fundraising for the beautiful Isla Rose and the John Lucas Foundation. Isla was born 30th September 2018. After a…
A new biotech startup in Dallas aims to speed the development of treatments for rare neurological diseases that disproportionately affect children. Taysha Gene Therapies, launched Tuesday with $30 million in seed financing, has partnered with UT Southwestern researchers whose mission is to eradicate genetic diseases…
Nationwide Children’s Hospital chooses Mayo Pujols, most recently of Novartis Pharmaceuticals, for its new biotech subsidiary Andelyn. As the new CEO for Andelyn Biosciences, a Nationwide Children’s Hospital-owned gene-therapy startup, Mayo Pujols will begin to assemble a diverse team of up to 200 scientists, biochemists,…
1st Patient Dosed in Trial of Gene Therapy, OTL-201, for Sanfilippo Type A Orchard Therapeutics announced the dosing of a first patient in a clinical trial evaluating its investigational cell-based gene therapy OTL-201 for Sanfilippo syndrome type A. OTL-201’s safety, tolerability, and efficacy is being investigated in the open-label, Phase 1/2…
Luk Vandenberghe, a gene therapy researcher at Massachusetts Eye and Ear and at Harvard Medical School, is spearheading an effort to develop a COVID-19 vaccine that uses an adeno-associated viral (AAV) vector to deliver genetic instructions for the SARS-CoV-2 spike protein into human cells. AAV…
Rare diseases are by their very nature obscure and unknown, falling through the cracks when it comes to research budgets and treatment development. But now, a study has shown they are more common than we think. New research shows that rare diseases are not that…
ZoomWalkathon.com Video Conferencing Lucas John Foundation is Actively Working to Save Children with Orphaned Rare Diseases FOR IMMEDIATE RELEASE May 2020 (Mesa, Arizona) – The family of Brandon and Chancee Culp in Mesa, Arizona, is fighting for the life of their two-year-old son, Lucas…
MESA, AZ (3TV/CBS 5) – A Mesa family is hosting a “Zoom the Room” virtual walkathon to raise awareness for NKH and money for gene replacement therapy research, which could be life-saving for their little boy. Click to Watch video of the story on AZ…
Submitted by Sarah Steel Having been featured in the Liverpool ECHO last year, Sarah Steel, a mother from Southport whose daughter, Isla, suffers from a rare metabolic disorder, is now renewing her daughter’s appeal in an effort to generate funding for scientists to explore gene therapy trials.…
You live in the shelter of the most high God. He alone is your place of safety, and we need to trust Him. Each morning we make sure we are “Psalms 91 Equipped” which declares protection and safety despite circumstances. We pray this Psalm over…