Rare and Orphan Diseases

Host: Mark Nolan Hill, MD

Guest: Randi Hagerman, MD

We are rapidly learning more about the genetic disorder Fragile X syndrome, and a series of conditions related to Fragile X. We're also seeing great progress in promising therapies under investigation. How will these therapies, along with other treatment strategies, improve the lives of children and families affected by Fragile X and related conditions? Who should supervise care, and should treatment for children be coordinated with care of potentially affected relatives? Dr. Randi Hagerman, professor and endowed chair of Fragile X research, and medical director of the Medical Investigation of Neurodevelopmental Disorders (MIND) Institute at the University of California, Davis School of Medicine, shares her expansive expertise on Fragile X syndrome and associated conditions with host Dr. Mark Nolan Hill. If a child is diagnosed with Fragile X, is genetic screening inevitably warranted for parents and grandparents?

Host: Mark Nolan Hill, MD

Guest: Randi Hagerman, MD

It's responsible for more than five percent of all cases of autism, and it's the most common cause of inherited mental retardation. But are we as familiar as we need to be with Fragile X syndrome, and a series of genetic conditions related to Fragile X? Host Dr. Mark Nolan Hill welcomes Dr. Randi Hagerman, professor and endowed chair of Fragile X research, and medical director of the Medical Investigation of Neurodevelopmental Disorders (MIND) Institute at the University of California, Davis School of Medicine, for a stimulating conversation about our rapidly expanding knowledge of these genetic conditions. How do the signs and symptoms manifest in children, and how does Fragile X impact our elder generations?

Host: Michael Greenberg, MD

Hypertrophic cardiomyopathy (HCM) is the leading cause of sudden death in young athletes. Joining host Dr. Michael Greenberg to discuss HCM is Dr. Martin Maron, director of the Hypertrophic Cardiomyopathy Center and co-director of cardiac MRI and CT at Tufts Medical Center. As an imaging specialist, Dr. Maron utilizes cardiac MRI and cardiac CT for the evaluation and treatment of patients. Discussing all aspects of HCM, Dr. Maron and Dr. Greenberg address this most common of all genetic cardiac diseases (occurring in 1/500 individuals), invasive and non-invasive treatment, and implications for HCM patients involved in sports.

Host: Maurice Pickard, MD

Guest: William Gahl, MD, PhD

Guest: Stephen Groft, PharmD

Some patients wait years for a definitive diagnosis. Using a combination of scientific resources and medical expertise at the National Institutes of Health (NIH), the Undiagnosed Diseases Program now provides a place for physicians to refer patients when a diagnosis eludes them. In this segment, host Dr. Maurice Pickard, speaks with Dr. William Gahl, director of the Undiagnosed Diseases Program, and Stephen Groft PharmD, director of the Office of Rare Diseases at the NIH, about program logistics and resources, and the referral process for doctors.

Host: Bruce Japsen

Guest: Jeffrey Aronin, MBA

It seems most drug-makers are making their biggest sales to doctors by pushing medicines for common conditions whether they be statins for cholesterol or proton pump inhibitors for heartburn. But drugs can actually take on blockbuster significance for specialized treatment groups where treatment options are limited. Jeffrey Aronin, president and CEO of Ovation Pharmaceuticals Inc. tells the Chicago Tribune's Bruce Japsen about his company's efforts to build blockbuster franchises by treating rare conditions.

Host: Bruce Japsen

Guest: Jeffrey Aronin, MBA

Not all drug companies are in pursuit of billion-dollar blockbuster drugs taken by millions of Americans. In fact, there are drugs treating rare conditions that wouldn't be sold on the market if not for a landmark piece of legislation known as the Orphan Drug Act, now 25 years old. Jeffrey Aronin, president and CEO of Ovation Pharmaceuticals Inc. tells the Chicago Tribune's Bruce Japsen about the silver anniversary of the Orphan Drug Act, its benefits and its future potential.

Host: Leslie P. Lundt, MD

Guest: James Levenson, MD

Alcohol abuse is a common concern for many patients afflicted with chronic pain conditions. What are we learning about alcohol use specific to patients with sickle cell anemia? Is alcohol perhaps more help than hindrance? Dr. James Levenson, professor of psychiatry, medicine and surgery at the Virginia Commonwealth University School of Medicine, shares his expertise in sickle cell disease with host Dr. Leslie Lundt.

Host: Mark Nolan Hill, MD

Guest: James Berenson, MD

Advances in multiple myeloma therapy are suppressing the dreaded expectations of a daunting disease. How are the latest treatment modalities presenting new avenues for cancer therapy? How can we work to improve our awareness of the changing face of multiple myeloma? Dr. James Berenson, founding president and chief executive officer of the Institute for Myeloma and Bone Cancer Research in West Hollywood, Calif., explores developments in multiple myeloma therapy that are improving prognoses for our patients. Dr. Mark Nolan Hill hosts.

Host: Mark Nolan Hill, MD

Guest: James Berenson, MD

Extended survival outcomes are becoming increasingly common after a diagnosis of multiple myeloma. What are some of the therapies responsible for these advances, and how far are we extending survival? Further, we’re learning that certain combination therapies are more effective for certain patients. How can we determine which patients are viable candidates for a specific treatment regimen? Dr. James Berenson, founding president and chief executive officer of the Institute for Myeloma and Bone Cancer Research in West Hollywood, Calif., probes these issues and more with host Dr. Mark Nolan Hill.

Host: Shira Johnson, MD

Guest: Brian Durie, MD

Noted hematologist Dr. Brian Durie is interviewed by host Dr. Shira Johnson on the role of environmental toxins in the development of multiple myeloma (MM). Dr. Durie discusses the increased incidence of MM in firefighters who participated in rescue and cleanup efforts after the terrorist attacks on 9/11, as well as the changing demographics of the disease.