Targeting the Toughest Diseases

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.   Featured guests: John Killian [http://ppmd.convio.net/site/TR/Global/General?px=1002778&pg=personal&fr_id=5403] 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 [https://www.parentprojectmd.org/dr-mena-scavina-joins-ppmd-as-certified-duchenne-care-center-program-advisor/]. Jon Rey-Hastie is the Co-Founder of Pathfinders Neuromuscular Alliance [https://www.pathfindersalliance.org.uk/meet-the-team/] Dr. David Altshuler [https://www.vrtx.com/our-company/leadership/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 [https://www.vrtx.com/our-science/pipeline/duchenne-muscular-dystrophy/].

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.    Featured guests: Quána Madison [https://quanamadison.com/bio] is an artist, mindfulness teacher, healing arts workshop facilitator, self-leadership coach, model and artist-philanthropist. She lives with chronic pain. Norm Buckley is the Scientific Director of the Michael G. DeGroote Institute for Pain Research and Care [https://healthsci.mcmaster.ca/pain-institute] at McMaster University.  David Altshuler [https://www.vrtx.com/our-company/leadership/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 [https://www.vrtx.com/our-science/pipeline/pain/] about Vertex's approach to targeting pain.

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 [https://www.11alive.com/article/about-us/team-bios/madison-carter/85-3ae8a4bd-56bf-4477-ab22-ef4d5dcbdffe] 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 [https://t1dfund.org/].  Felicia Pagliuca is the Disease Area Executive, Type 1 Diabetes, at Vertex Pharmaceuticals Doug Melton leads investigations into treatments for Type 1 Diabetes at Vertex Pharmaceuticals. He is renowned for his stem cell research at Harvard University [https://hsci.harvard.edu/people/douglas-melton-phd]. Read more about Vertex's approach to targeting type 1 diabetes [https://www.vrtx.com/our-science/pipeline/type-1-diabetes/] Produced by Bloomberg Media Studios [https://www.bloombergmedia.com/studio/] and Vertex Pharmaceuticals. [https://www.vrtx.com/]

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.    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 [https://www.bidmc.org/research/research-by-department/medicine/nephrology/laboratories/david-friedman-md] and a Principal Investigator and Nephrologist at Beth Israel Deaconess Medical Center. He's researched and consulted for Vertex Pharmaceuticals. Janice Lea [https://med.emory.edu/departments/medicine/divisions/profile/?u=JLEA]  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. [https://www.vrtx.com/our-science/pipeline/apol1-mediated-kidney-disease/#:~:text=The%20Vertex%20approach&text=Research%20has%20also%20shown%20that,of%20APOL1%2Dmediated%20kidney%20disease] Produced by Bloomberg Media Studios [https://www.bloombergmedia.com/studio/] and Vertex Pharmaceuticals [https://www.vrtx.com/].

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.   Featured guests: Terri Booker is an attorney and sickle cell advocate based in Philadelphia, Pennsylvania. Dr. Isaac Odame [https://www.sickkids.ca/en/staff/o/isaac-odame/#:~:text=Isaac%20Odame%20is%20the%20Haematology,for%20Sick%20Children%20]  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 [https://www.sickkids.ca/en/]. Dr. David Altshuler [https://www.vrtx.com/our-company/leadership/david-altshuler/] is the Executive Vice President, Global Research and Chief Scientific Officer at Vertex Pharmaceuticals. William Hobbs is a Vice President leading clinical development into hematology at Vertex Pharmaceuticals. Dr. William Hobbs is the VP- Clinical Development, Hematology at Vertex Pharmaceuticals. Read more [https://www.vrtx.com/our-science/pipeline/sickle-cell-disease/] about Vertex's approach to targeting sickle cell disease