Author Interviews, Pediatrics, Surgical Research / 22.11.2019

MedicalResearch.com Interview with: Ben Wheeler,MB ChB(Otago) DCH PhD CCE FRACP Paediatrician, Associate Department of Women's and Children's Health (Dunedin) University of Otag MedicalResearch.com: What is the background for this study? Response: All tongues have a frenulum, which is a small band of tissue that helps connect them to the floor of the mouth. Tongue tie (or ankyloglossia) is when this frenulum causes restriction to the movement of the tongue, and can interfere with successful breastfeeding in infants. This may be improved with an operation to cut the frenulum of the tongue (frenotomy). Internationally, tongue-tie diagnosis and treatment has increased substantially (reported at over 10-15% in some locations). This has led to growing concerns of potential overtreatment. The surgical treatment is often discussed as a minor surgery with little risk, but there is growing awareness this may not be the case. There is a paucity of studies examining moderate to severe complications following frenotomy. Therefore we aimed to determine rates of moderate to severe complications of tongue tie procedures presenting to hospital-based paediatricians in New Zealand, and describe this population. (more…)
Author Interviews, Heart Disease, Surgical Research / 21.11.2019

MedicalResearch.com Interview with: Chayakrit Krittanawong, MD Section of Cardiology Baylor College of Medicine Houston, TX MedicalResearch.com: What is the background for this study? Response: Transcatheter Aortic valve Implantation (TAVI) has emerged as equally effective alternative to surgical aortic valve replacement (SAVR) to treat severe aortic stenosis (AS) in all risk groups. In particular, less is known about the heart failure (HF) patients who undergo TAVI. Whether certain subtypes of HF respond differently after TAVI remains a mystery. In this study, we sought to assess and compare the incidence and predictors of in-hospital mortality among patients with heart failure with preserved ejection fraction (HFREF) versus heart failure with preserved ejection fraction (HFPEF). (more…)
Author Interviews, JAMA, Smoking, Tobacco, Tobacco Research / 21.11.2019

MedicalResearch.com Interview with: Amy L. Nyman, MA Research Associate Georgia State University, School of Public Health Atlanta, GA 30303 Coauthors: Nyman, Huang, Weaver, Eriksen MedicalResearch.com: What is the background for this study? Response: Though the negative health consequences of smoking combustible cigarettes have been well-established and widely accepted, there has been significant public debate about the health effects of Electronic Nicotine Delivery Systems (ENDS) use, and the extent to which use of ENDS may present less health risk to smokers than smoking combustible cigarettes. Many tobacco control researchers and scientists believe that ENDS use is safer than combustible cigarette smoking, though recent news of an expansive outbreak of vaping-related lung illnesses and deaths has led to continued speculation. A prior study of perceived comparative harm of combustible cigarettes and ENDS revealed the proportion of US adults perceiving ENDS to be as harmful as, or more harmful than combustible cigarettes increased substantially between 2012 and 2017. Given recently proposed regulations and the escalating focus on the impact of ENDS use on youth in the past few years, our study assessed changes in perceived comparative harm of combustible cigarettes and ENDS among US adults since the prior study, between 2017 and 2018.  (more…)
Author Interviews, Biogen / 20.11.2019

MedicalResearch.com Interview with: Aaron Deykin MD Vice President, Late Stage Clinical Development Biogen  MedicalResearch.com: What data support the U.S. Food and Drug Administration’s (FDA) approval of VUMERITY™ (diroximel fumarate)? Response: The FDA approval of VUMERITY was based on a New Drug Application (NDA) submitted under the 505(b)(2) filing pathway. It included data from pharmacokinetic bridging studies comparing VUMERITY and TECFIDERA® (dimethyl fumarate) to establish bioequivalence, and relied, in part, on the FDA’s findings of safety and efficacy for TECFIDERA. The NDA submission also included interim exposure and safety findings from EVOLVE-MS-1, an ongoing, Phase 3, single-arm, open-label, two-year safety study evaluating VUMERITY in patients with relapsing-remitting multiple sclerosis (MS). Interim results from EVOLVE-MS-1 at the time of NDA submission included a low overall rate of VUMERITY treatment discontinuation due to adverse events (6.3 percent), and a rate of less than one percent of patients who discontinued VUMERITY treatment due to gastrointestinal (GI) adverse events. Additional exploratory efficacy endpoints in the ongoing EVOLVE-MS-1 study showed changes in clinical and radiological measures compared to baseline. (more…)
Author Interviews, Heart Disease / 20.11.2019

MedicalResearch.com Interview with: Matthew Budoff MD Professor of Medicine, UCLA Endowed Chair of Preventive Cardiology Lundquist Institute Torrance, CA 90502 MedicalResearch.com: What is the background for this study? Response: We present the nine-month interim analysis results from the EVAPORATE mechanistic study of Icosapent Ethyl, after benefits were seen with the REDUCE-IT Trial, demonstrating 25% event reduction. This trial was a serial multi-detector computed tomographic (MDCT) study to look at plaque progression between icosapent ethyl (4 gm/day) and matching placebo.  (more…)
Author Interviews, Cancer Research, HPV / 20.11.2019

MedicalResearch.com Interview with: Ashish Deshmukh, PhD, MPH Assistant Professor UTHealth School of Public Health Houston MedicalResearch.com: What is the background for this study? Response: Anal cancer is one of the six human papillomavirus associated cancers.  Rates of anal cancer are increasing in the US, but no prior study quantified the contemporary trends (i.e., increase in rates over time) in anal cancer incidence. It was unknown whether the rise is real or driven by increased screening in some high-risk populations. Incidence trends according to age and stage at diagnosis was also never comprehensively studied. Furthermore, it was unknown whether the rise in incidence has led to a rise in mortality. Our objective was to answer these questions. (more…)
Accidents & Violence, Author Interviews, PTSD, Surgical Research / 20.11.2019

MedicalResearch.com Interview with: Michael A. Vella, M.D., M.B.A. Assistant Professor of Surgery Division of Acute Care Surgery and Trauma University of Rochester School of Medicine and Dentistry MedicalResearch.com: What is the background for this study? Response: There is a lot of (appropriate) focus on the mortality statistics related to gun violence, but sometimes we forget about the large number of survivors of gunshot wounds.  We wanted to specifically look at the long term physical and mental health outcomes in this patient population. (more…)
Author Interviews, Heart Disease, Omega-3 Fatty Acids, Prostate Cancer / 20.11.2019

MedicalResearch.com Interview with: Jeffrey L. Anderson, MD FAHA FACC MACP Distinguished Research Physician Professor of Medicine with Tenure University of Utah School of Medicine MedicalResearch.com: What is the background for this study? Response: Omega-3 supplements are widely used for cardiovascular prevention. However, a study published in the Journal of the National Cancer Institute (105:1132, 2013) reported as an incidental finding in a plasma bank study that the risk of prostate cancer increased with increasing levels of docosahexaenoic acid (DHA) and trended to increase with eicosapentaenoic acid (EPA). (more…)
Author Interviews, OBGYNE / 19.11.2019

MedicalResearch.com Interview with: Cynthia Gyamfi-Bannerman, MD, MSc Vice Chair for Faculty Development Ellen Jacobson Levine and Eugene Jacobson Professor of OBGYN Director, Maternal-Fetal Medicine Fellowship Program Co-Director, CUMC Preterm Birth Prevention Center  MedicalResearch.com: What is the background for this study? Response: ​Delayed cord clamping (DCC) at delivery has been associated with neonatal benefit.  Specially, it decreases the likelihood of anemia.  It became widely recommended as a practice for all deliveries even though the literature showing benefit to the neonates was largely only for low risk women with vaginal deliveries.  In theory, DCC could result in increased blood loss during a cesarean due to the blood loss encountered when cutting into a gravid uterus. (more…)
Author Interviews, CMAJ, Thyroid Disease / 18.11.2019

MedicalResearch.com Interview with: Dr. Richard Birtwhistle, Professor Departments of Family Medicine and Community Health and Epidemiology Director of the Centre for Studies, Primary Care, Department of Family Medicine Queen’s University MedicalResearch.com: What is the background for this study? Response: Testing for thyroid dysfunction is a commonly done by primary care practitioners. While the Thyroid stimulating hormone (TSH) test is an easy blood test to perform results outside the normal range are often found and will revert to normal over time without treatment in patients without symptoms. We wanted to see if there was any clinical benefit to patients by screening for thyroid dysfunction.  (more…)
Author Interviews, Lipids, NEJM, Neurological Disorders, Stroke / 18.11.2019

MedicalResearch.com Interview with: Pierre Amarenco, MD Professor and Chairman Paris University, Paris, France INSERM Department of Neurology and Stroke Centre Bichat Hospital Paris, France MedicalResearch.com: What is the background for this study? Response: The rationale of the Treat Stroke to target trial was that after we published the SPARCL trial in 2006 (atorvastatin 80 mg/day vs placebo in patients with stroke) which showed a 16% relative risk reduction of recurrent stroke, we performed several pre specified and post hoc analyses, showing that in SPARCL patients randomized with "atherosclerotic disease" the risk reduction for the primary endpoint was much higher (33%), and in in patients achieving a LDL cholesterol of less than 70 mg/dL as compared to those achieving a LDL cholesterol 100 mg/dL or higher, the risk reduction was 28%. Therefore to confirm this findings, we designed the TST trial, which was an investigator initiated trial funded by the french ministry of health,  and enrolled patients with an ischemic stroke due to atherosclerotic stenosis and randomized them to either a target LDL cholesterol of less than 70 mg/dL or a target LDL cholesterol of 90 to 110 mg/dL. To achieve these goals, the investigators could use any statin available on the market, and titrate the dosage of the statin to get to the assigned target. They could also use ezetimibe on top of statin therapy if a high dosage of statin was not sufficient to get to the target level assigned by randomization. (more…)
Author Interviews, Dermatology, Gender Differences, Pediatrics / 17.11.2019

MedicalResearch.com Interview with: Markus Boos, MD, PhD Member of the Society for Pediatric Dermatology. Attending pediatric dermatologist Seattle Children's Hospital Assistant Professor in the Department of Pediatrics University of Washington School of Medicine  MedicalResearch.com: What is the background for this study? Response: Our understanding of the cutaneous health of sexual and gender minority (SGM) individuals (lesbian, gay, bisexual, transgender, queer, asexual, intersex, nonbinary, etc.) remains nascent. This dearth of understanding of the unique needs of SGM children is even more pronounced. This 2-part review article provides practical advice on how to best engage with young SGM patients and serve the distinct needs of this minority population, with a specific emphasis on dermatologic conditions. (more…)
Author Interviews, Cannabis, Heart Disease, Stroke / 17.11.2019

MedicalResearch.com Interview with: Rupak Desai, MBBS Research Fellow, Division of Cardiology Atlanta VA Medical Center MedicalResearch.com: What is the background for this study? Response: Amidst legalization of therapeutic and recreational use of marijuana/cannabis in the United States, cerebrovascular effects of marijuana use remain largely unknown, especially among young adults. We examined the association between cannabis use (18–44 years) among young adults and stroke events. The study analyzed pooled data from the Behavioral Risk Factor Surveillance System (2016–2017)—a nationally representative cross-sectional survey collected by the Centers for Disease Control and Prevention. Overall, 13.6% of total 43,860 young adults (18-44 years) reported using cannabis recently (in the last month), with 63.3% of them being men. Compared with nonusers, marijuana users were often younger, non-Hispanic white or black, and with some college education. (more…)
Author Interviews, Dermatology, JAMA, Melanoma / 17.11.2019

MedicalResearch.com Interview with: Jennifer M. Gardner, MD Clinical Assistant Professor of Dermatology University of Washington School of Medicine  MedicalResearch.com: What is the background for this study? Response: This study looked at age-specific differences of melanoma incidence in the United States. It was an observational study looking at population-based registry data extracted from the combined National Program of Cancer Registries-Surveillance Epidemiology and End Results United States Cancer Statistics (NPCR-SEER) database. The overall take home message from this study is that though melanoma incidence has continued to climb in the past decade for both men and women, most of the increase is seen in adults greater than age 40 years of age.  In contrast, melanoma incidence decreased in adolescents (ages 10-19 years of age) and young adults (ages 20-29) after peaking around 2004-2005. Melanoma is more common in males in older individuals (older than 50 years of age) but in younger individuals (<50 years of age), melanoma is more common in females.  According to a recently published JAMA-Otolaryngology paper by Bray and colleagues, there may be a subset of younger individuals where males are at a higher risk than females in regard to head and neck melanoma, and after that study was published we noted this to be true in our numbers, as well (we didn’t publish this in our study), further identifying a possibly “at risk” demographic within the younger age groups in addition to young women. (more…)
Abuse and Neglect, Electronic Records, Yale / 15.11.2019

MedicalResearch.com Interview with: Edward R. Melnick, MD, MHS Assistant Professor of Emergency Medicine Program Director, Yale-VA Clinical Informatics Fellowship Program Principal Investigator, EMBED Trial Network Yale School of Medicine New Haven, CT 06519  MedicalResearch.com: What is the background for this study? Response: We know that physicians are frustrated with their EHRs and that EHRs are a driver of burnout. This is the first study to measure these issues nationally. We included a standardized metric of technology from other industries (System Usability Scale, SUS; range 0-100) on the AMA’s 2017 physician burnout survey. This metric has been used in >1300 other studies so we can compare where the EHR’s usability is to other everyday technologies. We are also able to measure the relationship between physicians’ perception of their EHR’s usability and the likelihood they are burned out. (more…)
Author Interviews, Breast Cancer, JAMA, Mammograms / 15.11.2019

MedicalResearch.com Interview with: Dr. Elham Kharazmi, MD, PhD Co-Leader, Risk Adapted Prevention (RAD) Group Division of Preventive Oncology National Center for Tumor Diseases (NCT) German Cancer Research Center (DKFZ) Germany MedicalResearch.com: What is the background for this study? Response: Breast cancer is the most common cancer in women worldwide and the second leading cause of cancer death in American women, exceeded only by lung cancer. Available evidence suggests that implementation of a screening program can decrease breast cancer mortality. Reductions in breast cancer mortality in Europe over the past two decades have been associated at least in part with the implementation of screening programs. Screening enables the detection of tumors at an early stage, when more treatment options are feasible and most effective. However, screening is associated with substantial risks, such as over-diagnosis, false-positive results, and physical and psychological harms, particularly when large numbers of women with low risk are frequently screened. (more…)
Allergies, Author Interviews / 15.11.2019

MedicalResearch.com Interview with: Christopher M. Bland, Pharm.D., FCCP, FIDSA, BCPS Clinical Associate Professor Clinical and Administrative Pharmacy University of Georgia College of Pharmacy Clinical Pharmacy Specialist St. Joseph’s/Candler Health System Savannah, GA MedicalResearch.com: What is the background for this study? Response: Nearly 10% of the United States population self-report a penicillin allergy. However 90% of these allergies are found to be false upon reconciliation which includes patient interview, graded challenge, direct challenge, or penicillin skin testing. This is crucial as patients labeled with a penicillin allergy often receive more expensive antibiotics that additionally cause more adverse effects. While reconciling penicillin allergies is an important antimicrobial stewardship goal, resources are often limited in various healthcare settings to accomplish several of these endeavors. Our study evaluated the use of pharmacy students to serve as patient interviewers to aid in reconciling penicillin allergies. (more…)
Author Interviews, Biomarkers, Melanoma, Ophthalmology / 15.11.2019

MedicalResearch.com Interview with: Dr. Mitchell Stark, B.App.Sc (Hons), PhD NHMRC Research Fellow The University of Queensland Diamantina Institute Woolloongabba, QLD MedicalResearch.com: What is the background for this study? Response: Uveal nevi (moles) mimic the appearance of uveal melanoma and their transformation potential cannot be definitively determined without a biopsy. Moles or naevi in the eye are common but can be difficult to monitor because changes to their shape or colouring can’t always be seen as easily as on the skin. As naevi are difficult to biopsy, they are usually “monitored” at regular intervals. If there is a melanoma in the eye, then outcomes are poor for people if their cancer spreads to the liver. This study aimed to identify a “biomarker” that could be measured in patients’ blood that could be used as an early indicator of melanoma formation (from a mole) or progression to other body sites.  (more…)
Author Interviews, Technology, Vaccine Studies / 15.11.2019

MedicalResearch.com Interview with: Sandra Crouse Quinn, PhD Professor and Chair, Department of Family Science Senior Associate Director, Maryland Center for Health Equity School of Public Health University of Maryland College Park, MDSandra Crouse Quinn, PhD Professor and Chair, Department of Family Science Senior Associate Director, Maryland Center for Health Equity School of Public Health University of Maryland College Park, MD  MedicalResearch.com: What is the background for this study? Response: Millions of Americans use Facebook (FB) for a variety of purposes, including seeking health information. However, it is difficult for FB users to discern what is credible and scientifically sound information on the platform.  Facebook ads also focus on health issues including vaccination, which is labelled by FB as an issue of national importance.  We were the first team to conduct a research study examining the FB Ad Archive and analyzed ads that contained vaccine content. (more…)
Author Interviews, Rheumatology / 14.11.2019

MedicalResearch.com Interview with: Benjamin Nowell, Ph.D. Director of Patient-Centered Research CreakyJoints, Principal Investigator of ArthritisPower  MedicalResearch.com: What is the background for this study? Response: Patient-reported outcomes (PROs) are important indicators of treatment effectiveness, but little is known about which PRO measures that patients find the most important to track for their disease management and to evaluate treatment effectiveness and health outcomes. In this study, we used the ArthritisPower Research Registry to evaluate which PROs patients with rheumatological conditions voluntarily selected to understand their experience of disease.  (more…)
Allergies, Author Interviews, Cancer Research, Pediatrics / 13.11.2019

MedicalResearch.com Interview with: Sairaman Nagarajan, MD Clinical Fellow at State University New York (SUNY) Downstate Medical Center College of Medicine MedicalResearch.com: What is the background for this study? Response: The impetus for this study came from our previous research linking asthma, hay-fever and overall cancer diagnoses using the CDC’s National Health Interview Survey database. The division of Allergy and Immunology at SUNY Downstate has also conducted two pilot studies on the relationship between parental cancer and childhood asthma in Brooklyn’s population; one from Lutheran Medical Center focusing on Hispanics and Asian patients, and the other on African-American and Afro-Caribbean patients. (more…)
Author Interviews, Education, Gender Differences, JAMA / 13.11.2019

MedicalResearch.com Interview with: Shannon Ruzycki, MD, MPH, FRCPC, (she/hers) General Internist & Clinical Lecturer Department of Medicine Department of Community Health Sciences Cumming School of Medicine University of Calgary MedicalResearch.com: What is the background for this study? Response: After hearing about the National Academies of Science, Engineering, and Medicine 2018 report to congress on sexual harassment of women in academia, our Department Head asked myself and Dr. Aleem Bharwani to study experiences of gender inequity or equity in our Department. We conducted an in-depth, 18-month mixed methods study of women and men in our Department, including semi-structured interviews and survey. (more…)
Author Interviews / 13.11.2019

MedicalResearch.com Interview with: Magdalena Cerdá DrPH Associate Professor and Director Center for Opioid Epidemiology and Policy Department of Population Health NYU Langone School of Medicine  MedicalResearch.com: What is the background for this study? Response: Legalization of recreational marijuana use is increasingly enacted across US states and across the world. In the US, 11 states and D.C. have legalized recreational use of marijuana, while 33 states and D.C. have legalized medical use of marijuana. In light of these changes, we need to know the impact that recreational marijuana legalization has on marijuana use. To our knowledge, our is the first study to look at the impact that recreational marijuana legalization has had on both use and cannabis use disorder among different age groups.  (more…)
Allergies, Author Interviews, Electronic Records / 13.11.2019

MedicalResearch.com Interview with: Sonam Sani MD Allergy & Immunology Fellow NYU Winthrop Hospital MedicalResearch.com: What is the background for this study? Response: Penicillin allergy label removal is becoming more common. Studies have shown that while 10% of the general population report an allergy to penicillin, after testing only 1% truly have an allergy. Allergists have the ability to evaluate patient’s for penicillin allergy by performing skin tests and oral challenges. However, even when people test negative for penicillin allergy, they still face barriers to having the label removed. We are noting more and more that despite having negative testing, upon further encounters, our patients still have their penicillin allergy label. (more…)
Author Interviews, Brigham & Women's - Harvard, Cost of Health Care, Rheumatology / 13.11.2019

MedicalResearch.com Interview with: Natalie McCormick, Ph.D. Post-Doctoral Research Fellow Clinical Epidemiology Program Division of Rheumatology, Allergy, and Immunology, Massachusetts General Hospital Department of Medicine, Harvard Medical School Arthritis Research Canada  MedicalResearch.com: What is the background for this study? Response: Biologic disease-modifying anti-rheumatic drugs (bDMARDs) have improved the health and productivity of many people living with moderate-to-severe inflammatory rheumatic diseases. They are also among the highest-spend drugs in the USA, with substantial out-of-pocket costs that pose barriers to treatment initiation and adherence. To understand the drivers of ongoing bDMARD spending growth, and effective ways of containing costs, we analysed drug spending data for all bDMARD claims in Medicare Part D, Part B fee-for-service, and Medicaid over 2012 to 2016, isolating the impact of changes in drug prices from changes in utilisation.  (more…)
Author Interviews, Opiods, Rheumatology / 12.11.2019

MedicalResearch.com Interview with: Raveendhara R. Bannuru MD, PhD, FAGE Director, Center for Treatment Comparison and Integrative Analysis (CTCIA) Deputy Director, Center for Complementary and Integrative Medicine (CCIM) Asst Professor of Medicine, Tufts University School of Medicine Asst Professor of Clinical & Translational Science, Sackler School of Graduate Biomedical Sciences Division of Rheumatology, Tufts Medical Center Boston, MA MedicalResearch.com: What is the background for this study? Response: Given the current controversy regarding the use of opioids in chronic pain, we wanted to delve deeper into the efficacy and safety profiles of oral opioid drugs in osteoarthritis patients. Temporal assessments like ours can reveal peak periods of efficacy, and can provide clinicians with a blueprint for optimal durations of treatment regimens. With respect to subgroup analyses based on strength of opioid binding affinity, we sought to explore currently held paradigms that strong opioids may be useful for the treatment of severe pain, and to specifically assess their relevance in OA populations. Knowledge of the relative efficacy and safety profiles of strong versus weak opioids can give clinicians the information they need to weigh benefits and harms of specific subgroups of opioids. (more…)
Author Interviews, Rheumatology / 11.11.2019

MedicalResearch.com Interview with: Kelly Gavigan, MPH Manager, Research and Data Science CreakyJoints  MedicalResearch.com: What is the background for this study? Response: Over the past fifteen years, there have been significant improvements in quality of life among people living with rheumatic and musculoskeletal disease with the introduction of biologics and targeted therapies. However, despite a variety of treatments to try, patients often seek non-pharmacological alternative and complementary treatments, such as marijuana for medical use (MMU), to help manage their condition and symptoms. MMU is becoming increasingly available in the United States as different states legalize it under specific circumstances. Legal or not, according to a survey conducted by CreakyJoints using the ArthritisPower Research Registry (n=1,059 participants), people with arthritis are trying marijuana for medical use.  (more…)
Author Interviews, Pain Research, Rheumatology / 11.11.2019

MedicalResearch.com Interview with: Féline PB Kroon MD Department of Rheumatology Leiden University Medical Centre LUMC · MedicalResearch.com: What is the background for this study? Response: Hand osteoarthritis (OA) is a prevalent joint condition that causes pain, functional disability, and decreased quality of life, for which patients frequently consult health-care providers. Symptoms usually fluctuate over time, with episodes of joint swelling and erythema. Evidence from previous studies has shown that inflammation plays an important role in the disease, being an important predictor for pain and radiographic damage progression. Therefore, we hypothesized that inflammation could be a treatment target in OA, and we investigated this using prednisolone, a potent anti-inflammatory drug. The aim of the HOPE study was to investigate the clinical efficacy and safety of a six-week course of prednisolone 10 mg daily in patients with painful hand OA who had evidence of synovial inflammation. (more…)
Author Interviews, Gout, Kidney Disease, Rheumatology, Transplantation / 10.11.2019

MedicalResearch.com Interview with: Megan Francis-Sedlak, PhD Director of Medical Affairs Horizon Therapeutics Lake Forest, Illinois MedicalResearch.com: What is the background for this study? Response: The prevalence of gout is more than ten-fold greater among patients who have undergone a kidney transplant than the general population as post-transplant medications to prevent organ rejection can contribute to increased uric acid levels. Overall studies have shown this can lead to higher rates of uncontrolled gout among this vulnerable population with organ transplants. While we have seen higher mortality rates for patients who have received a kidney transplant with uncontrolled gout compared to kidney transplant patients without uncontrolled gout, we wanted to evaluate the impact of gout on transplant-related complications to better inform patient care and treatment approaches.  (more…)
Author Interviews, Genetic Research, Kidney Disease, Mineral Metabolism, Pediatrics / 10.11.2019

MedicalResearch.com Interview with: Tracy McGregor, MD MSCI Alnylam Pharmaceuticals Cambridge, Massachusetts  MedicalResearch.com: What is the background for this study?
  • Lumasiran is an investigational RNA interference (RNAi) therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1). PH1 is a rare life-threatening disease in which a enzymatic deficiency in the liver results in pathologic overproduction of oxalate, often leading to recurrent kidney stones and a progressive decline in kidney function, which typically culminates in end-stage renal disease (ESRD).Patients with ESRD are at a risk of systemic oxalosis, with oxalate depositing throughout the body, including the eyes, skin, bones, and the heart. Complications associated with ESRD and/or systemic oxalosis can be fatal. For patients with ESRD treatment options are limited and include intensive dialysis as a bridge to a dual liver/kidney transplant, highlighting the unmet need for new treatment options. 
(more…)