Allergies, Asthma, Author Interviews, Dermatology, Pediatrics / 10.03.2020

MedicalResearch.com Interview with: Marcella Aquino, M.D. Hasbro Children's Hospital Department of Pediatrics Division of Allergy & Immunology Associate Professor of Pediatrics   Daphne Koinis-Mitchell PhD Professor (Research) in the Departments of Psychiatry and Human Behavior and Pediatrics Warren Alpert Medical School of Brown University Providence, Rhode Island 02903   MedicalResearch.com: What is the background for this study? Response: Urban minority children with asthma are at increased risk for sleep loss and poorer sleep quality secondary to socio-contextual stressors (poverty, stressors of urban living) and the underlying challenges related to following possibly complex asthma treatment regimens. Atopic dermatitis (AD) is very frequently seen in children with asthma and increases the risk for poor quality sleep, for example difficulty falling asleep, awakenings during the night, difficulty awakening in the morning, and/or daytime sleepiness.  (more…)
Author Interviews, Brain Injury, Exercise - Fitness, Pediatrics / 05.03.2020

MedicalResearch.com Interview with: Robert F. Heary, M.D. Co-Director, Reynolds Family Spine Laboratory Director, Spine Center of New Jersey Professor, Rutgers New Jersey Medical School Newark, New Jersey MedicalResearch.com: What is the background for this study? Response: This article was written to assess the relative danger versus safety of youth football.  As this is a hot-button topic in the world of neurosurgery and neurology, we decided to look into this issue. In a suburban town, middle school football players were studied.  They wore helmets with accelerometers mounted inside the hemet to measure how many hits the player absorbs and the magnitude of the force behind the hits.  Also, soft “guardian caps” we worn over the outside of the helmets during practices. For all football activities (practices and games), the helmets were worn and data were accumulated.  In addition, specialized coaching related to safe tackling techniques was provided. (more…)
Accidents & Violence, Author Interviews, Brigham & Women's - Harvard, JAMA, Pediatrics / 03.03.2020

MedicalResearch.com Interview with: Hooman Azad First author and a 3rd year medical student Feinberg School of Medicine Northwestern University Eric Fleegler, MD MPH FAAP Senior author and Pediatric Emergency Physician Boston Children’s Hospital Assistant Professor of Pediatrics and Emergency Medicine Harvard Medical School MedicalResearch.com: What is the background for this study? Response: Pediatric firearm violence is a public health crisis. The firearm fatality rate has increased by >50% over the past 10 years. Over our 26-year study period (1991-2016), 13,697 children under the age of 15 died at the hands of a firearm. Laws have been employed to try to reduce these deaths, and Child Access Prevention (CAP) laws, which aim to hold parents liable for the safe storage of their firearms, were passed in 25 states between 1989-2000. No new state passed a CAP laws after the year 2000. Child Access Prevention laws come in two flavors – recklessness laws that hold firearm owners liable for directly providing firearms to a minor, and negligence laws that hold the firearm owner liable for the unsafe storage of firearms with variability in how storage is defined and what penalties are imposed. (more…)
Author Interviews, Nutrition, Pediatrics, Sugar / 03.03.2020

MedicalResearch.com Interview with: Lori Spruance PhD Assistant Professor, Public Health College of Life Sciences Brigham Young University MedicalResearch.com: What is the background for this study? Response: I was interested in studying this phenomenon after I attended a research conference presentation where the presenter was describing results from a study where they found that children between ages 6-11 who were physically active drank more sugar-sweetened beverages than children in that age group who were not physically active. The presenter suggested that they believed this was due to youth sports and Gatorade consumption. I was really intrigued by the fact that kids were getting physical activity, but had really high sugar-sweetened beverage intake. Prior to the study, I did some qualitative interviews with parents of children involved in youth sports. During these interviews, parents discussed the “snack culture” and the shame they sometimes feel being the “healthy parent”. I really wanted to investigate further what was happening. (more…)
Addiction, Author Interviews, JAMA, Pediatrics / 02.03.2020

MedicalResearch.com Interview with: Karl Alcover, PhD Postdoctoral Research Associate Behavioral Health Innovations Washington State University MedicalResearch.com: What is the background for this study? Response: It has been a public health focus to prevent early exposure to drugs. Our paper shows that the average age of initiation of drug use among adolescents and young adults has been increasing from 2004 to 2017. We found that 12 of 18 drugs (including alcohol and tobacco products) had statistically increasing average ages of initiation. To our knowledge, no studies have documented these findings. We think this is great news because delaying initiation of drugs prevents early exposure, which we know is associated with various long-term negative health outcomes. Also, these promising trends may serve as initial evidence that prevention strategies, especially those that focus on adolescents and young adults, are working. (more…)
Allergies, Author Interviews, CMAJ, Environmental Risks, Pediatrics / 20.02.2020

MedicalResearch.com Interview with: Jaclyn Parks, B.Sc. Health Sciences M.Sc. Health Sciences Candidate | Faculty of Health Sciences Simon Fraser University Burnaby, B.C MedicalResearch.com: What is the background for this study? Response: Childhood asthma is a major public health concern, and many researchers are interested in determining environmental and modifiable exposures in early life so that we can recommend preventative measures. The findings of our study add to the understanding of which exposures in early life may be important to the development of childhood asthma and allergies and allows us to identify specific areas of intervention for parents and other stakeholders involved in protecting children’s health. (more…)
Author Interviews, Gastrointestinal Disease, Pediatrics / 14.02.2020

MedicalResearch.com Interview with: Dr. Mohammadali Khan Mirzaei Institute of Virology Helmholtz Zentrum München Neuherberg, Germany  MedicalResearch.com: What is the background for this study? Can you briefly explain what is meant by a bacteriophage? Response: Child stunting a severe growth impairment, globally affecting about 1 in 5 of children. The correlation between altered gut microbiota and stunting is already known. In contrast to what we know about the link between altered gut bacteria and stunting, the role of phages was not explored. Phages are the bacterial viruses that match the number of bacterial cells by a 1:1 ratio in the human gut. They are central to the biogeochemistry of most ecosystems by driving bacterial physiology, diversity, and abundance. Therefore, we expect a significant role for them in the human gut. (more…)
Author Interviews, Dental Research, Environmental Risks, Pediatrics / 12.02.2020

MedicalResearch.com Interview with: R. Constance Wiener, DMD, PhD Associate Professor West Virginia University School of Dentistry MedicalResearch.com: What is the background for this study? Response: Perfluoroalkyl and polyfluoroalkyl substances (PFAS) are chemical groups that have had a wide variety of uses due to their ability to their ability to repel water and stains. They might be found in food packaging, water-repellant clothing and carpeting, paints, fire-fighting foam, and water, for example. Although many are no longer manufactured in the United States, PFAS persist in the environment as they do not readily break down. Adverse health effects have been speculated especially for low birthweight babies, immunological effects, certain cancers and thyroid hormone disruption.1 With these considerations, we hypothesized that there may be an association of PFAS with tooth development and subsequent dental caries (cavities). (more…)
Asthma, Author Interviews, Brigham & Women's - Harvard, NEJM, Pediatrics, Vitamin D / 10.02.2020

MedicalResearch.com Interview with: Augusto A. Litonjua, M.D., M.P.H. Professor - Department of Pediatrics, Pulmonology Interim Chief - Department of Medicine , Pulmonary Diseases and Critical Care Professor - Department of Medicine , Pulmonary Diseases and Critical Care University of Rochester MedicalResearch.com: What is the background for this study? Response: Vitamin D deficiency and insufficiency is prevalent worldwide. Prior observational studies have shown that low vitamin D levels have been associated with the development of asthma. Animal studies have reported that antenatal vitamin D is important for lung development in utero. Thus, we conducted a randomized, double-blind, controlled trial of vitamin D supplementation in pregnant women to see if we could prevent the development of asthma and wheezing illnesses in young children. The initial report of the trial results showed that children born to mothers in the vitamin D supplementation arm had lower risks for developing either asthma or recurrent wheezing episodes over the first 3 years, but this was not statistically significant (p=0.051)(Litonjua et al. JAMA 2016). (more…)
Author Interviews, OBGYNE, Pediatrics / 04.02.2020

MedicalResearch.com Interview with: Andrea M. Tilstra Doctoral Candidate, Department of Sociology Population Program, Institute of Behavioral Science University of Colorado Boulder MedicalResearch.com: What is the background for this study? Response: Average U.S. birth weight declined across the 1990s and 2000s, and this has puzzled most researchers. We investigate this and find that the increases in cesarean deliveries and induction of labor between 1990 and 2013 resulted in a shift in the gestational age distribution of U.S. births. We find that births are less likely to occur at gestational weeks 40+ and much more likely to occur between weeks 37-39. Additionally, results from our simulations show that if U.S. rates of cesarean deliveries and labor induction had not increased over time, then average birth weight would have increased.   (more…)
Author Interviews, ENT, JAMA, Obstructive Sleep Apnea, Pediatrics / 03.02.2020

MedicalResearch.com Interview with: Dr. Jonathan R. Skirko, MD , MHPA, MPH Assistant professor Division of Pediatric Otolaryngology University of Utah Health  MedicalResearch.com: What is the background for this study? Response: Obstructive sleep apnea is a common problem that that impacts the lives of many people. Understanding treatment effectiveness is important and Health-State Utility is a standardized way of assessing quality of life.  Before this study, we didn't have a way of measuring quality of life in this population in this important way. You have to accurately measure something before you can improve it. (more…)
Abuse and Neglect, Author Interviews, JAMA, Pediatrics, Tobacco / 30.01.2020

MedicalResearch.com Interview with: Hongying (Daisy) Dai, PhD Associate Professor Department of Biostatistics | College of Public Health University of Nebraska Medical Center Omaha, NE MedicalResearch.com: What is the background for this study? Response: E-cigarette use increased dramatically from 11.7% to 27.5% for high school students and from 3.3% to 10.5% for middle school students during the periods of 2017 - 2019. In September 2018, the Food and Drug Administration (FDA) warned that youth e-cigarette use is reaching an epidemic proportion. Exposure to secondhand aerosol (SHA) from e-cigarettes is not harmless as e-cigarettes aerosol contains nicotine and potentially harmful substances, including carbonyl compounds, TSNAs, heavy metals, and glycols. This study analyzed the 2015-2018 National Youth Tobacco Survey (NYTS) and the main findings are:
  • The prevalence of secondhand aerosol exposure significantly increased from 25.6% in 2017 to 33.2% in 2018 (p<.001) after being stable during 2015 and 2017 (25.2% vs. 25.6%, p>0.05). The increase of SHA exposure from 2017 to 2018 was observed across socio-demographic groups.
  • Among never tobacco users in 2018 NYTS, students who reported secondhand aerosol exposure (vs. no) had higher odds of susceptibility to use e-cigarettes (38.8% vs. 21.0%) and cigarettes (30.7% vs. 21.2%) and higher odds of reporting exposure to e-cigarette marketing.
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Author Interviews, JAMA, Pediatrics, Weight Research / 29.01.2020

MedicalResearch.com Interview with: Dr. Carol Chelimo PhD Research Fellow Dept. of Paediatrics, School of Medicine University of Auckland MedicalResearch.com: What is the background for this study? Response: New Zealand has the third highest prevalence of obesity among Organisation for Economic Co-operation and Development countries. Pediatric obesity is associated with development of cardiovascular risk factors in later life, such as type 2 diabetes, hypertension, dyslipidemia, and metabolic syndrome. Antibiotic exposures in early life may affect weight by altering the gut microbiota, potentially increasing the risk of childhood obesity. The overall aim of this research was to examine whether repeated antibiotic exposure by age 48 months is associated with higher body mass index (BMI) at age 54 months. Specifically, it evaluates whether the number, timing (age), and type of antibiotic exposures are associated with a higher body mass and an increased likelihood of overweight and obesity. This work incorporates antibiotic exposure during pregnancy (more…)
Author Interviews, Blood Pressure - Hypertension, Duke, Pediatrics / 22.01.2020

MedicalResearch.com Interview with: Yuichiro Yano MD Assistant Professor in Family Medicine and Community Health Duke University  MedicalResearch.com: What is the background for this study? Response: The blood pressure (BP) guideline in the US recommend using an “average” of multiple BP measurements over time for screening for and management of high BP in young adults. While it is well known that BP varies across visits, that “variability” (i.e., visit-to-visit blood pressure variability) is dismissed as a random fluctuation in the clinical setting. Little is known regarding the clinical relevance of visit-to-visit blood pressure variability over time in young adults. (more…)
Author Interviews, Depression, Dermatology, Pediatrics / 21.01.2020

MedicalResearch.com Interview with: Dr. Jonathan L. Silverberg MD PhD MPH Director of Clinical Research and Contact Dermatitis Associate Professor of Dermatology George Washington University School of Medicine and Health Sciences Washington, DC  MedicalResearch.com: What is the background for this study? Response: We previously found that children from single parent families, and unsafe or unsupportive neighborhoods are more likely to have atopic dermatitis. Parents in these settings may experience greater psychosocial distress and higher rates of depression in the post-partum period and beyond. As such, we sought to understand the relationship of maternal depression with atopic dermatitis in their children. (more…)
Author Interviews, Dermatology, Mental Health Research, Pediatrics / 15.01.2020

MedicalResearch.com Interview with: Lucia Diaz, M.D., is chief of pediatric dermatology, dermatology residency associate program director and assistant professor of medicine and pediatrics at Dell Medical School. She is also co-director of the dermatology-rheumatology combined clinic at Dell Children’s Medical Center. Sasha Jaquez, Ph.D. is a pediatric psychologist at Dell Children's Medical School/Dell Children's Medical Center and specializes in seeing children with chronic medical illness, including skin disorders.     MedicalResearch.com: What is the background for this study? Response: Trichotillomania (TTM) can be an extremely disabling chronic condition that impacts the psychosocial development of children. It is classified by the Diagnostic and Statistical Manual of Mental Disorders (DSM-5) as an obsessive-compulsive disorder, where a person recurrently pulls out hair from any region of their body resulting in hair loss. Recognizing this disorder and being informed of treatment options allows medical providers to correctly diagnose and intervene early in the disease course. We reviewed the psychosocial impacts of pediatric trichotillomania and the current evidence-based interventions used in the population.  (more…)
Accidents & Violence, Author Interviews, Brain Injury, JAMA, Pediatrics, UCSF / 08.01.2020

MedicalResearch.com Interview with: Benjamin N. Breyer MD, MAS, FACS Associate Professor Departments of Urology and Epidemiology and Biostatistics University of California, San Francisco Vice-Chair of Urology Chief of Urology, Zuckerberg San Francisco General Hospital and Trauma Center Director, UCSF Male Genitourinary Reconstruction and Trauma Surgery Fellowship MedicalResearch.com: What is the background for this study? Response: There has been a large increase in upright scooter usage among adults as a mode of transportation. It's convenient for commuters and may encourage greater use of public transit leading to less car traffic in cities. (more…)
Author Interviews, JAMA, Opiods, Pediatrics / 06.01.2020

MedicalResearch.com Interview with: Rachel H. Alinsky, MD, MPH Adolescent Medicine and Addiction Medicine Fellow Division of General Pediatrics and Adolescent Medicine Johns Hopkins University School of Medicine MedicalResearch.com: What is the background for this study? Response: We know that over 4,000 adolescents and young adults between the ages of 15-24 are dying from an opioid overdose every year. Nonfatal opioid overdose has been identified as a potential touchpoint with the healthcare system when individuals can be drawn into treatment, yet very little is known about health care use following opioid overdose in youth. We were interested in figuring out the extent to which adolescents and young adults are receiving evidence-based treatment after an opioid overdose. (more…)
Author Interviews, Brigham & Women's - Harvard, JAMA, OBGYNE, Pediatrics, Weight Research / 02.01.2020

MedicalResearch.com Interview with: Elvira Isganaitis, M.D., M.P.H. Pediatric Endocrinologist, Joslin Diabetes Center Assistant Professor of Pediatrics, Harvard Medical School Boston, MA 02215 MedicalResearch.com: What is the background for this study? Response: The concept that a mother's nutrition prior to and during pregnancy is important for health outcomes in the offspring is now well accepted. For example, women intending to get pregnant must take prenatal vitamins, and are encouraged to attain a healthy weight before conception. However, much less is known about how a father's nutritional status may influence childhood health outcomes.  Based on studies in animals, exposure to undernutrition, high-fat diet, or stressful experiences in fathers can result in increased risk of obesity and diabetes in the offspring. These effects are mediated in part by epigenetic mechanisms (i.e. changes in gene expression due to differences in DNA methylation, histones, or other non-genetic mechanisms). (more…)
Author Interviews, Critical Care - Intensive Care - ICUs, Mental Health Research, Opiods, Pediatrics / 23.12.2019

MedicalResearch.com Interview with: Megan Land, MD, PGY 6 Pediatric Critical Care Medicine Fellowship Emory University School of Medicine MedicalResearch.com: What is the background for this study?
  • Much of the research on the opioid crisis has focused on the impact to adults; however, children and adolescents in the US are also negatively affected by the opioid epidemic.
  • The percentage of children admitted to a pediatric intensive care unit increased over the study period as the clinical effects of the opioid ingestions increased in severity.
  • The primary intent of opioid ingestions was suspected suicide attempts in adolescents resulting in increasing admissions to a psychiatric hospital.
  • Opioids associated with the highest odds of needing an intervention in an intensive care unit were methadone, fentanyl, and heroin. 
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Author Interviews, Cannabis, JAMA, Pediatrics / 20.12.2019

MedicalResearch.com Interview with: Hongying (Daisy) Dai, PhD Associate Professor Department of Biostatistics | College of Public Health University of Nebraska Medical Center  MedicalResearch.com: What is the background for this study? Response: E-cigarette use increased significantly from 2017 to 2019 among U.S. adolescents, and marijuana and other substances besides can be used in e-cigarettes. Meanwhile, restrictions on marijuana use have been relaxing and social acceptability of marijuana use is shifting among youth. This study analyzed 38,061 middle and high school students from the 2017 and 2018 National Youth Tobacco Survey.  (more…)
Author Interviews, JAMA, Opiods, Pediatrics, University of Michigan / 19.12.2019

MedicalResearch.com Interview with: Kao-Ping Chua, MD PhD Assistant Professor, Department of Pediatrics Susan B. Meister Child Health Evaluation and Research Center University of Michigan MedicalResearch.com: What is the background for this study? Response: Opioids are frequently prescribed to adolescents and young adults aged 12-21 years – in a recent study, 1 in 8 patients in this population were prescribed opioids during the year. At the same time, almost 30% of the 3000 opioid-related overdose deaths in 2016 among adolescents and young adults involved prescription opioids. Given the frequency of opioid prescribing and the risk of overdose, it is important to understand how to prescribe opioids safely to adolescents and young adults. However, there have been few studies that examine which opioid prescribing patterns are associated with prescription opioid overdose in adolescents and young adults. Prior studies examining these patterns have focused on older adults, particularly U.S. Veterans, so the generalizability of these findings to younger populations is unclear. (more…)
Author Interviews, Autism, Genetic Research, Nature, Pediatrics / 10.12.2019

MedicalResearch.com Interview with: Dr. Stephen Scherer, PhD, FRSC Senior Scientist, Genetics & Genome Biology Director, The Centre for Applied Genomics SickKids Hospital Toronto MedicalResearch.com: What is the background for this study? Response: One of the most common questions we get from parents with a child with autism is, "what is the likelihood of having a second or third child with autism, and what is the chance others in our family will have kids with autism?". To help provide answers to these questions, we started the infant (or baby) siblings study ten years ago. Families having an older sibling with a diagnosis of autism were invited to enroll their next born for assessment and following to see if they also developed autism, and what the likelihood of that happening was. Biological samples like blood, and DNA from blood, were also collected and tested.  (more…)
Author Interviews, Brigham & Women's - Harvard, Global Health, JAMA, Pediatrics, Vaccine Studies / 09.12.2019

MedicalResearch.com Interview with: Emily Parker Hyle, M.D. Assistant Professor of Medicine Massachusetts General Hospital MedicalResearch.com: What is the background for this study? Response: We found that many children who were planning to travel internationally were eligible for MMR vaccination prior to departure but often did not receive it - especially if they were aged 6 months to 6 years. That is because most children do not routinely receive their first dose of MMR till 12-15 months of age and their second dose of MMR till 4-6 years of age. However, ACIP recommendations are different for children who are traveling internationally. The risk of being infected with measles is much higher outside of the US, so it is recommended that children older than 1 year have had 2 MMR vaccinations and that children 6-12 months receive 1 MMR vaccination prior to travel. MMR vaccination is a safe and effective way to greatly reduce the risk of measles infection.  (more…)
Author Interviews, Education, ENT, Hearing Loss, JAMA, Pediatrics / 30.11.2019

MedicalResearch.com Interview with: Dr. Carlijn M. P. le Clercq, MD Speech and Language Pathology, Pediatrics, Otolaryngology Erasmus MC, Rotterdam MedicalResearch.com: What is the background for this study? Response: Recently, more reports have been publishes about the prevalence of slight to mild hearing loss among children in the general population. These studies showed that slight hearing loss is common, also within our population-based cohort in the Netherlands. Slight hearing loss is often scored as “not relevant”. We wanted to examine if we could find associations of hearing thresholds in children with performance scores in everyday life. (more…)
Author Interviews, Brigham & Women's - Harvard, Cannabis, JAMA, Pediatrics / 26.11.2019

MedicalResearch.com Interview with: Bertha K. Madras PhD Director, Laboratory of Addiction Neurobiology Psychobiologist, Substance Use Disorders Division, Basic Neuroscience Division Professor of Psychobiology, Department of Psychiatry Harvard Medical School MedicalResearch.com: What is the background for this study? Parent use of marijuana is rising, and I wondered whether this could be associated with offspring use of specific substances and across several substances
  • Several fathers have confided in me that they used marijuana to bond with their sons. They became horrified after witnessing their sons progress to using other drugs, especially heroin.
  • In general, living with a parent using substances or having substance use disorders is an explicit risk for use of substances among young offspring. Yet, few studies have directly examined whether parental marijuana use elevates the risk for opioid misuse among adolescent and young adults living with parents.  Most importantly and to the best of our knowledge, none of the existing research  simultaneously explored frequency of  parental marijuana use and whether it related to adolescent and young adult offspring’s marijuana, tobacco, alcohol use, and opioid misuse.
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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, 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…)
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, 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. 
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