Popular on eTradeWire
- Great Kids Books You Never Knew Existed - 147
- Paige Dimakos named CEO of The Draft Network
- Readiness Associates forms strategic partnership with CloudOak
- Consultant and Publicist to Present At New Year New You - 2 Day Teleconference
- Casual workers left out to dry by Broxbourne Council during pandemic
- Burstcoin Joins BPSAA as New Alliance Member
- TVS Flashback Network.Com Showcases A Dozen More Color Classic TV Shows on WatchYour.TV
- Former UK Environment Minister backs revolutionary plastic that solves pollution issues
- New Book Release: The Mom in Me, Stories and Practical Advice from Moms Who have Survived Parenting
- SmartDesk 4 Sneak Peak: Pricing and Release Date Announced
Similar on eTradeWire
- CKMB Monoclonal Antibodies (Affinity Pure)
- 1α-Hydroxy Vitamin D4 (Highly Pure)
- Grand Opening of The SNW Outlet on Saturday December 5th at the St Nicholas Centre, Sutton
- Jessica Genel, MS, RD, Awarded National Bariatric Board Certification
- Nashaat Tawfik, MBA B.Sc. Pharmacy, CBE, Awarded Appointment to COTE Advisory Board
- Journey Medical Corp Announces New Journey with the Experts Video Featuring Diane Berson, MD
- 78-year-old CS:GO Champ DieHardBirdie Talks About the Health Benefits of Gaming in Fighting Dementia
- Medication Adherence Expertise Center and Pill Connect help patients with TB take their medication
- Exeter Orthodontics Opens New Location, Offering Braces in Springfield
- Finding Inner Peace During the Holiday Season
Rare Disease Foundation Signs $1.4 Million Contract To Produce Gene Therapy Vector For Hunter Syndrome Clinical Trial
"This is a critical step towards our goal to develop an effective gene therapy product to treat individuals with MPS II," said Dr. Kim McBride, the clinical principal investigator of the project. "This project reflects two decades of collaborative efforts of Dr. Haiyan Fu at Nationwide Children's Hospital and Dr. Joseph Muenzer at University of North Carolina at Chapel Hill."
In 2013, Project Alive began working with Dr. Fu to see if her previous preclinical gene therapy research for a similar disease, Sanfilippo Syndrome (MPS III), could be developed in Hunter Syndrome. That call began a symbiotic relationship between a researcher who knew the science and parents most familiar with the disease, which accelerated the then ongoing collaborative research project of Drs. Fu and Muenzer.
More on eTradeWire News
- TVS Music Network.Com to Produce 26 More Episodes of Senior Prom in their Philadelphia Studio
- Isabella Johnston, aka The Intern Whisperer, is a GUINNESS WORLD RECORD Participant
- The Me-Suite Podcast Ranked 'Best Of' 2020
- Research infographic shows how vital trade shows are to global business
- Single Operated Newsletter and The Spiritual Movement
Affecting approximately 2,000 people worldwide, Hunter Syndrome is a rare, genetic condition resulting in the lack of the enzyme iduronate-2 sulfatase. Without that enzyme, cellular waste builds up throughout the body and causes progressive loss of physical, and in most cases, mental function. The clinical trial targets the most common, severe form of Hunter Syndrome, causing progressive neurological decline and a life span in the early teens. To date, no gene therapy or gene editing has been performed in a patient with cognitive regression, which represents approximately two-thirds of Hunter patients.
"With the generous support from Project Alive and many other family foundations we have made pivotal progress since 2014," said Dr. Fu. "This has led to significant advancement in our MPS II gene therapy research towards a clinical trial."
"Last week, we also received FDA approval of our investigational new drug (IND) application, which will allow us to proceed with clinical trials for this gene therapy product in MPS II patients," added Dr. Kim McBride who will lead the trial at NCH. "We truly appreciate the continuous efforts and philanthropy of Project Alive and other patient foundations to sponsor this trial."
To support the clinical trial, Project Alive has been raising funds from public donations, grants and fundraisers across the United States. They have also released a documentary series about Hunter Syndrome that has received significant media attention including on national television shows such as "The Doctors."
More on eTradeWire News
- Tobu Railway Transports Travelers from Tokyo's Shitamachi Downtown to Historic Nikko, where the New FUFU Nikko Onsen Resort Opened in October 2020
- Federal Primary Election Update From Chance For President
- "Colosseum" by Richard Valanga is published
- Celebrity Master Fitness Trainer Author Solomon Pratt announced as New Beneficience.com PR Client
- Black Car Everywhere Limousine & Car Service
Recently the foundation raised over $100,000 on Giving Tuesday to support the vector production for the clinical trial. Project Alive continues to seek major donors who want to have a lasting impact on this devastating disease, as well as on the healthcare system since current treatments for Hunter Syndrome run approximately $500,000 per patient per year.
"We are excited to move forward in our efforts to save these children's lives," notes Melissa Hogan, president of Project Alive, Hunter Syndrome expert, and parent to a ten-year-old with Hunter Syndrome. "But now we need the funding to catch up with the science and our mission."
To find out more about Hunter Syndrome or to support the mission of Project Alive, visit ProjectAlive.org.
- Donna S. Bellinger "The GodMother" in Business Sales entertains Gratitude on Entrepreneurship LIVE
- Candlestick Queens Event Appeals to Women of Color Who are Interested in Forex Trading
- Megan Thee Stallion Got "Body" Flow From Female Rapper Aynjewl Faycc
- Illustrating History With Tea!
- PicPocket-Labs, Inc. announces wholly-owned subsidiary Stories.inc - launches Stories.app
- Humanitarian Paul Rusesabagina's Nov 27 Hearing: Kidnapped, Illegally Detained & Tortured by Rwanda
- The Carbonell Awards Announces Major Operational Changes in 2021
- Rosalinda Books on Writing and Making Movies
- Rise Up Quarantine Winner Releases New Single Worldwide
- Voyagers Toastmasters Presents How to Conduct a Webinar
- Matthew Brickman Explains the Benefits of Online Dispute Resolution Throughout The 2020 Holiday Season
- Local Car Dealerships And Baseball Team Donate 1000 Turkey Dinners & Trimmings
- Mounts Botanical Garden Celebrates December with Fun, Festive & Furry Events
- Elliot on Hollywood and how he's evolved as an actor
- Freelance Work May Save You From The Recession
- How to reduce busy calendaring work to 1 minute per day
- NJ MED launches its six-annual National High School Graduation Pledge Campaign
- Unique and Uplifting Pet-Inspired Subscription Box from Dog Mom Lifestyles
- Mind Shift 2020, New Book by Joshua S. Kangley, Provides Inspiration
- Identity: The Podcast A Christian's Identity Feat. Pastor Taylor