Targeting Sickle Cell Disease

John Killian’s son was diagnosed with Duchenne muscular dystrophy (DMD) as a toddler. Until recently, children with DMD were not expected to live beyond their teens. 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. Featured guests:John Killian is the proud father of Sam Killian, who lives with DMD.Mena Scavina is a Neurologist and Clinical Director at Nemours Children's Health and a Clinical Care Team Advisor at Parent Project Muscular Dystrophy.Jon Rey-Hastie is the Co-Founder of Pathfinders Neuromuscular AllianceDr. David Altshuler is the Executive Vice President, Global Research and Chief Scientific Officer at Vertex Pharmaceuticals. Alison McVie-Wylie is a Vice President leading the Duchenne muscular dystrophy research team at Vertex Pharmaceuticals.Read more about Vertex's approach to targeting Duchenne muscular dystrophy. 

Damian Sanchez, (Damian J to music fans) is a Miami-based jazz musician. A car accident landed him in the hospital with multiple fractures to his leg. Once there, he faced the difficult decision of whether or not to take the pain medication that was offered to him. With patients like Damian in mind, 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 differently.Produced by Bloomberg Media Studios and Vertex Pharmaceuticals. Featured guests:Damian Sanchez is a Miami based jazz musician. He is the founder of the Damian J Foundation.Norm Buckley is the Scientific Director of the Michael G. DeGroote Institute for Pain Research and Care at McMaster University. David Altshuler is the Executive Vice President, Global Research and Chief Scientific Officer at Vertex Pharmaceuticals.Paul Negulescu is a renowned cystic fibrosis researcher and now the Senior Vice President leading the team researching pain at Vertex Pharmaceuticals.Read more about Vertex's approach to targeting pain at VRTX.com 

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.  Featured guests:Madison Carter is an investigative journalist with 11Alive News, an NBC affiliate in Atlanta, GA. She's living with type 1 diabetes. Aaron Kowalski is the President and CEO of JDRF. Felicia Pagliuca is the Disease Area Executive, Type 1 Diabetes, at Vertex PharmaceuticalsDoug Melton leads investigations into treatments for Type 1 Diabetes at Vertex Pharmaceuticals. He is renowned for his stem cell research at Harvard University. Read more about Vertex's approach to targeting type 1 diabetesProduced 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 African Americans carry. Dr. Ogo Egbuna leads clinical development for the team researching APOL1-mediated kidney disease at Vertex where they’re investigating a potential therapy to target its underlying cause. Alonzo Mourning is a paid spokesperson for Vertex Pharmaceuticals.Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.  Featured guests:Alonzo Mourning is a retired NBA player, the Vice President, Player Programs for the Miami Heat, an advocate for kidney disease research, and a beneficiary of a kidney transplant.David Friedman is an Associate Professor of Medicine, Harvard Medical School and a Principal Investigator and Nephrologist at Beth Israel Deaconess Medical Center. He's researched and consulted for Vertex Pharmaceuticals.Janice Lea  is a Professor of Medicine and Clinical Director of Nephrology at Emory University School of Medicine.Ogo Egbuna is Vice President, Clinical Development at Vertex Pharmaceuticals Read more about Vertex's approach to targeting kidney disease.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, and they are investigating multiple approaches to address the unmet needs that patients face.Produced by Bloomberg Media Studios and Vertex Pharmaceuticals. Featured guests:Terri Booker is an attorney and sickle cell advocate based in Philadelphia, Pennsylvania.Dr. Isaac Odame  is the Haematology Section Head in the Division of Haematology/Oncology and the Medical Director of the Global Sickle Cell Disease Network at the Centre for Global Child Health at The Hospital for Sick Children.Dr. David Altshuler is the Executive Vice President, Global Research and Chief Scientific Officer at Vertex Pharmaceuticals. Dr. William Hobbs is the VP- Clinical Development, Hematology at Vertex Pharmaceuticals.Read more about Vertex's approach to targeting sickle cell disease  

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. Featured guests:Boomer Esiason is a retired NFL quarterback, Co-Chairman of the Boomer Esiason Foundation and Broadcaster with CBS SportsGunnar Esiason is the Executive Vice President, Strategy and Advocacy at the Boomer Esiason Foundation and lives with cystic fibrosis.Chinedu Nwokoro is a Consultant in Paediatric Respiratory Medicine at The Royal London Children's Hospital, Barts Health NHS Trust David Altshuler is the Executive Vice President, Global Research and Chief Scientific Officer at Vertex Pharmaceuticals. Read more about Vertex's approach to targeting cystic fibrosis.

Targeting the Toughest Diseases looks at some of humanity’s most challenging diseases and how Vertex, a Boston-based biotech company, is using innovative tools, methods, and a unique philosophy to search for treatments and cures. Produced byBloomberg Media Studios and Vertex Pharmaceuticals.The first episode drops October 10th. Subscribe today.