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May 7, 2024 We are deeply saddened to share that a patient participating in our Phase 2 DAYLIGHT study for Duchenne muscular dystrophy has passed away suddenly. On behalf of everyone at Pfizer, we extend our sympathies to his family, friends, and those closest to his care. We do not yet have complete information and are actively working with the trial site investigator to understand what happened. The patient received the investigational gene therapy, fordadistrogene movaparvovec, in early 2023. Initial dosing for the placebo-controlled, randomized Phase 3 CIFFREO trial, which utilizes a crossover trial design, was also completed in 2023. We have decided to pause dosing associated with the cross-over portion of CIFFREO, and we are working with regulators and the independent external Data Monitoring Committee as we learn more about this event. Other than this pause in dosing, trial activities are continuing as scheduled. Both the DAYLIGHT and CIFFREO trials are investigating fordadistrogene movaparvovec in patients with DMD, in different age cohorts: DAYLIGHT in boys 2 years to less than 4 years of age, and CIFFREO in boys 4 to less than 8 years of age. Beyond CIFFREO, the dosing pause does not apply to other ongoing trials in the fordadistrogene movaparvovec program as dosing has been completed in those studies. The safety and well-being of the patients in our clinical trials remains our top priority, and we are committed to sharing more information with the medical and patient community as soon as we can. We are also aware that many in the patient community are hopeful about the potential benefit of fordadistrogene movaparvovec for the treatment of DMD, and we will continue to collect data from our trials to evaluate its ability to address this disease.  As always, if you have questions please do not hesitate to reach out. Sincerely, The Pfizer DMD gene therapy team

Over the past decade, in a quest to fight the deadly disease that ails his son, Manalapan’s Jim Raffone has trekked to Mount Everest base camp , built a record-setting Lego chain , appeared on a special-edition Father’s Day card , and rung the closing bell of the New York Stock Exchange. Next up: Holding a half-marathon on Route 9. It took several years, but Raffone has lined up approvals for the 13.1-mile race, dubbed “NJ Run 4 Hope,” which will pass through five municipalities on the morning of Sunday, Oct. 6. Most of the course is along Route 9 South, which will shut down and reopen gradually as the race unfolds. Proceeds will fund a clinical trial for Duchenne muscular dystrophy — a rare and fatal genetic disorder that causes progressive muscle weakness. Jim’s son James (Jamesy) Raffone was diagnosed with Duchenne at age 4 . He’s now 15 and a freshman at Howell High School. The life expectancy for Duchenne patients is generally in the mid-20s. “Right now time is against us, so with this happening and with the help of all the mayors, it’s been a godsend,” Jim Raffone said. “I can’t thank everyone enough.” This is believed to be the first half-marathon or marathon taking place mostly along Route 9. The race will begin at All American Ford in Old Bridge and proceed south through Marlboro and Manalapan, with a loop through Freehold Borough before ending at iPlay America in Freehold Township. “We’re going to have a huge celebration and a business expo at iPlay,” Raffone said. JAR of Hope, the nonprofit the Raffone family launched in 2014, has been granted approval by the U.S. Food and Drug Administration to begin clinical trials on an investigational drug to treat Duchenne. The trials will be conducted by researchers at the University of Florida. Raffone said he’s hoping to raise $1 million with a field of at least 1,000 runners. The clinical study is expected to cost about $2 million; JAR of Hope has been fundraising for it since the study was approved in 2021. “I would be blown away if we could get 3,000 people (to race),” Raffone said. “We can change the course of this disease by funding a clinical trial with one race.” For more information on NJ Run4Hope, visit www.njrun4hope.com , email info@njrun4hope.org or call 732-414-6670. Jerry Carino is community columnist for the Asbury Park Press, focusing on the Jersey Shore’s interesting people, inspiring stories and pressing issues. Contact him at jcarino@gannettnj.com. 

Jim shares his journey from learning his only son has Duchenne Muscular Dystrophy to running 10 marathons including running over 300 miles from Massachusetts to New Jersey, doing push up around the world and climbing Mt Everest, all to bring awareness to and helping find a cure for this terminal disease.

Nebraska man joins fight against Duchenne muscular dystrophy

Originally posted on Newsbreak

Will you accept this challenge Millstone Twp. & Upper Freehold Regional School Districts? 

-- Study meets primary endpoint with secondary and exploratory endpoints showing consistency with primary endpoint -- -- Data reinforces previously observed safety profile for Givinostat; treatment in boys with DMD continues to show a good tolerability profile -- -- The company is planning to discuss the potential for marketing application submission with regulatory authorities for Givinostat in DMD -- -- Results were presented at the Annual PPMD Conference on June 25, 2022 -- 

Hypoxia as a Modulator of Cardiac Disease in Duchenne muscular dystrophy by Dr. DeWayne Townsend Jamesy is one of the first children with Duchenne muscular dystrophy to undergo HBOT treatment. Through this study, his marked improvements have led to the first ever hyperbaric pre-clinical trial to be hosted by the University of Minnesota. The launch of the HBOT research is a primary focus of our current fundraising efforts. The findings may provide the vital information needed to prove the clinical efficacy of oxygen therapy, and could lay the groundwork to make this treatment available to all children suffering from Duchenne. The goal of the HBOT study is to enable these children to walk for as long as possible, until a cure can be found.

Equal justice for Charities: allow Charities to sell raffles tickets on-line in NJ