Popular on eTradeWire
- Mona Clayton, RN Launches Mix & Mentor emPOWER Hour Global Tour at Hotel 50 Bowery 114
- Even Neutral Switzerland Can't Stomach Philip Morris
- Justices Free Up Funding For Trump's Wall
- Variety – The Children's Charity of Wisconsin CEO on Modern Living with kathy ireland®
- Home2 Suites by Hilton Abilene Celebrates Topping Off
- Goldberg Brothers ships new folding barn door hardware
- Shuvu Banim Announces Israeli Rabbi Eliyahu Merav Video Statement of Support for Rabbi Berland
- Radio Vet Connie Birth returns to The Spin Awards as the Red Carpet host
- Lyn Ross of Institut' Dermed Prize Winner in the 2019 Skin Games Compassionate Category
- Macy's Caves Over "Fat Shaming," But Maybe It Works
Similar on eTradeWire
- Chicago Children's Museum Awarded National Science Foundation Grant
- Buck Wild Prepares to Launch New Products; Venom Milk, Dung Beetle Bar, Chinchilla Chips & More
- LDC and ISAR Bioscience Team up to Develop Novel Regenerative Therapies
- MMS Holdings and Sage IT Celebrate 10-Year Relationship with Deep Focus on Intelligent Automation and Bot Development
- Assay.Works and ISAR Bioscience Team up to Develop Novel Regenerative Therapies
- Brendan Sammon joins The Logit Group Inc
- Sales Education Foundation Awards $15,000 in 2019 Sales Research Grant Program
- ITSMA Announces Finalists for the 2019 Marketing Excellence Awards
- Sequoia at the forefront of ocean color remote sensing, releases impactful new technology!
- Leukemia Research Foundation Funds 12 New Investigators
Kim Stephens, president of Project Alive, updated the Hunter Syndrome community on the progress of the study and the organization's fund-raising efforts during a Facebook Live message on Wednesday evening -- May 15, MPS Awareness Day. See her message here: https://www.facebook.com/projectalive/videos/20...
The safety studies, a necessary first step, are needed to begin the gene therapy drug trial for a promising cure for Hunter Syndrome.
"What this means is we've gone through checks to make sure that the vector is safe in the animal models," Stephens said referring to vehicle by which the desired gene is delivered to the cells during the therapy being tested. "After the safety study is complete, a report of the findings will be submitted to the FDA. This will bring us another step closer to moving forward with the actual study of the gene therapy drug."
More on eTradeWire News
- Pearson Edexcel bags The Word Masala Champion of the Diaspora Poetry Award
- VORAGO Technologies Announces Space-saving ARM® Cortex®-M0 (MCU) NVM for Extreme Rad
- Don Barnhart's Hypnomania Residency Gets Extended
- Tour Lennar's 10 Model Homes at Glen Loma Ranch Now Open Daily
- Two Ways Home, starring Tanna Frederick and Tom Bower an official Women Texas Film Fesitval selection will be screening on Saturday, August 17th
Project Alive is a grassroots organization formed by parents whose children suffer from Hunter Syndrome, a rare degenerative disease that affects mainly boys and limits life expectancy to the early teen years in the most severe cases. The organization works to raise awareness for Hunters Syndrome, provides support for Hunter Syndrome families and raises funds for research to find a cure. The non-profit has raised a total of $2.35 million and funded the preclinical work and vector manufacturing for the trial set to be conducted at Nationwide Children's Hospital in Columbus, Ohio. That is just $150,000 shy of the funding needed for the full clinical trial.
"We needed a cure yesterday, but we're optimistic we will be able to move things along to the next step very soon," Stephens said, whose son Cole was diagnosed with Hunter Syndrome in 2012 at two and a half years old. The founders of Project Alive, the world's leading Hunter Syndrome research and advocacy foundation, initiated their own gene therapy research for the disease when there was none. They contacted and collaborated with researchers and have been funding this trial from its preclinical stages to its IND approval from the FDA to its current stage. They will continue to advocate for families affected by Hunter Syndrome as well as act as a resource for the rare disease community once the trial begins.
More on eTradeWire News
- AccuSource, Inc. Ranks on the 2019 Inc. 5000 With Three-Year Revenue Growth of 92 Percent
- Join WOMBA at the 5th Annual SHIEC Conference
- Executive Director Search Announced
- Skyscanner for Dummies: 3 Ways to book your next Skyscanner Tickets
- Car Title Loans Can Take Off the Pressure When Sudden Lay Offs Sting, says Car Cash Loans
Project Alive is poised to serve the Hunter Syndrome community by continuing to fund and design research, assist industry in the space, and bring new insights into the scientific community about this rare disease. Project Alive continues to work closely with the National MPS Society and the other Hunter Syndrome groups with the goal of saving and improving the lives of those affected by the disease. Plans are in the works to start a rare disease registry for Mucopolysaccharidosis or MPS. Hunter Syndrome is a form of Mucopolysaccharidosis known as MPSII.
Project Alive is a powerful voice for children and adults with Hunter Syndrome, bringing together families and advocates with researchers, industry, and regulators. Project Alive funds promising curative research, assists researchers and industry with designing research studies for our community, and advocates for the most effective and efficient system of clinical research, evaluation, and approval. Through its innovative campaigns and grassroots efforts, Project Alive has made significant advances in public awareness about Hunter Syndrome and its symptoms, the need for early diagnosis, and available treatments and clinical trials.
- Experienced Financial Advisor Chooses Cutter & Company in Launching Her Own Independent Firm
- IoT Data Logging with Expert Devices
- Work 365 Sync for Self-Service and Provisioning Automation
- Celebrity Charity Football Tournament at Seaford FC in Sussex for 4 Charities
- Bravo's Married To Medicine Reality Star Draws Breast Cancer Survivors to Atlanta's City Hall
- Gaslowitz Frankel Attorneys Recognized in 2020 Edition of Best Lawyers in America
- Jazz Across the Water, Bordum and Rhodes
- Kelley Kronenberg's Alison Verges Walters Named a Top Women Leader in Accounts Receivable
- Disruptive Startup Award @ Stanford
- The Beautiful Day Un Dia Bonito Magazine inspired by the Show hitting the Tri-State region
- West Virginia Writer Highlighted in Greenbrier Valley Quarterly
- Refereum rewards Dauntless players over USD $3,000 in prizes
- Wisconsin Author Debuts A Fresh and Fun Plot in New YA Book
- Smiles for Innocents: 102 Exonerees to Receive Free Dental Care
- Metro Building and Remodeling Group LLC Named by REMODELING Magazine to its 2019 Big50 Class of America's Top Remodelers
- We Sell Restaurants Sells Zaatar of Plantation, Florida
- Utica Zoo welcomes 2 newborn Red Panda cubs
- Climate Change-Global Warming Proven Solutions to Reducing Carbon Pollution -ECOFuelMax
- Southern Ocean Chamber Association presents summer donation to Parker's Army
- GA Gov visits Atlanta area community health center saving eyesight of under served Georgians