Most parents wouldn’t be thrilled with the idea of their kids getting hooked up to an IV bag filled with trillions of viruses. But for Melanie Hennick, whose son, Connor, has Duchenne muscular dystrophy, it was an opportunity she hoped would change his life. “We knew this wasn’t a cure,”...
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Washington. DC – The FDA has denied approval of Theratechnologies’ F8 formulation of tesamorelin, which the Canadian biotech was proposing to reduce excess abdominal fat in patients with HIV and lipodystrophy, the company announced Wednesday. In its Complete Response Letter, the regulator pointed to issues regarding tesamorelin’s chemistry, manufacturing and controls,...
Greenwich, CT – The Fibrolamellar Cancer Foundation is pleased to announce that it has funded a new study directed by Roshni Dasgupta, MD of the Cincinnati Children’s Hospital Medical Center (CCHMC). Dr. Dasgupta is a leader in surgical oncology and the chair of the Pediatric Surgical Oncology Research Collaborative (PSORC)....
Port Elizabeth, South Africa – Fibrous dysplasia is a benign tumour-like condition in which normal bone is replaced by structurally poor fibro-osseous lamellar bone. It occurs as a result of an early embryonic postzygotic activating mutation of the GNAS gene which manifests clinically in a wide spectrum of disease. Fibrous...
Tuberculosis should not be a death sentence in 2009. It wasn’t for me nearly 70 years ago after I contracted the disease in the Terezin concentration camp in what was then Czechoslovakia. But today, nearly 2 million people die from TB each year, an unconscionable number of lives lost to...
As we enter what I consider to be the most wonderful time of the year, I reflect on why this season of giving is so magical. Does it really have anything to do with the gifts under the tree, the parties, or the Christmas lights? Many of us fill our December...
KUSA – Beauty is only skin deep. Everyone knows that. But our actions don’t always reflect that understanding. For a clear picture of what that means we turn to the story of a teenager, yes a teenager. Seventeen-year-old Kelley Sperry is surrounded by peers that many of us might view...
Eddie Ndopu, an award-winning disability activist from South Africa, and one of 17 United Nations advocates for the Sustainable Development Goals, lives with spinal muscular atrophy, and faces many difficulty daily challenges. Ahead of International Day of Persons with Disabilities, on 3 December, Mr. Ndopu discusses how he has overcome...
West Des Moines, Iowa – Born in 2013, identical twins Kenzie and Kaylie Morell were hitting some, if not all, of their normal infant and toddler milestones—until they began to slip back from them. When Kenzie was around 15 months old, she started to regress. Kaylie followed a year and...
Trent Hills – How many five-year-olds can say they’ve thrown the ceremonial first pitch at a Blue Jay game the night the team’s ace pitcher Roy Halladay is honoured as Sportsperson of the Year, or shared the stage with international pop star John Mayer at a concert in Toronto? Isaac...