Targeting the Toughest Diseases Vertex Pharmaceuticals

Before John Killian's toddler son was diagnosed with Duchenne muscular dystrophy (DMD), doctors told parents to simply take their children home and love them. The disease weakens the body's muscles and cognitive functions, which can lead to premature death. John’s son is now college-bound, thanks to the help of medical advancements that target the symptoms of the disease. Geneticist Dr. Alison McVie-Wylie and the research team at Vertex hope to move past only treating the symptoms of DMD by addressing the disease at its root cause.

Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.

Quána Madison is one of the millions of Americans living with chronic pain. Dressing herself or even showering can be excruciating. Dr. Paul Negulescu and the research team at Vertex are thinking differently about pain – they’re investigating potential non-opioid based treatment options that aim to target pain where it starts. Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.

Madison Carter is a fearless investigative reporter. She takes no day for granted because she lives with type 1 diabetes (T1D). Dr. Doug Melton is a world-renowned stem cell researcher and distinguished fellow at Vertex – and his now grown children, Sam and Emma, live with T1D. Along with scientists like Dr. Felicia Pagliuca, who leads Vertex’s research into T1D, he’s been on a quest to help Madison, Sam, Emma, and others like them by investigating the disease at a cellular level.

Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.

NBA great Alonzo Mourning returned home from the Sydney Olympics after winning the gold medal feeling ill. He surprisingly was diagnosed shortly after with kidney disease. Scientists have discovered his type of kidney disease is linked to having genetic variants of the APOL1 gene – ones that 13% of people with African ancestry carry. Dr. Ogo Egbuna leads clinical development for the team researching APOL1-mediated kidney disease at Vertex where they’re investigating a small molecule therapy to target its underlying cause.

Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.

Terri Booker’s body burns with pain. She’s one of the 100,000 Americans living with sickle cell disease – a blood disorder that can cause severe pain, organ failure, and even premature death. The genetic condition came about to protect against malaria. Dr. William Hobbs leads clinical development for sickle cell disease at Vertex – their investigational research into sickle cell disease uses cutting-edge technology.

Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.

NFL quarterback Boomer Esiason’s career high came just days before his son was diagnosed with cystic fibrosis (CF) – a genetic disease characterized by thick and sticky mucus that interferes with breathing, digestion and the body’s ability to fight infections. Esiason now dedicates his free time to raising awareness to fund the search for a cure. We explore how Vertex’s Chief Scientific Officer, Dr. David Altshuler, and the research team at Vertex have spent the last 20+ years researching the underlying cause of the disease – and their work is not yet finished.

Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.