Author Interviews, Biogen, Neurological Disorders, Pharmaceutical Companies / 30.04.2020
Spinal Muscular Atrophy: Biogen Enrolls First Patient in Higher-Dose SPINRAZA® (NUSINERSEN) Study
MedicalResearch.com Interview with:
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Dr. Ferguson[/caption]
Toby Ferguson, M.D., Ph.D.
Vice President, Head Neuromuscular Development Unit
Biogen
MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by SMA, who is primarily affected and incidence?
Response: Spinal muscular atrophy (SMA) is a rare, genetic, neuromuscular disease characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA. It is a leading genetic cause of infant mortality; however, people of all ages are impacted by the disease.
More than three years ago, SPINRAZA (nusinersen) became the first FDA-approved treatment option for SMA.
The DEVOTE study, which recently treated its first patient, is designed to evaluate the safety and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved for the treatment of SMA. The Phase 2/3 randomized, controlled, dose-escalating study will be conducted at approximately 50 sites around the world and aims to enroll individuals of all ages with SMA.
Dr. Ferguson[/caption]
Toby Ferguson, M.D., Ph.D.
Vice President, Head Neuromuscular Development Unit
Biogen
MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by SMA, who is primarily affected and incidence?
Response: Spinal muscular atrophy (SMA) is a rare, genetic, neuromuscular disease characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA. It is a leading genetic cause of infant mortality; however, people of all ages are impacted by the disease.
More than three years ago, SPINRAZA (nusinersen) became the first FDA-approved treatment option for SMA.
The DEVOTE study, which recently treated its first patient, is designed to evaluate the safety and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved for the treatment of SMA. The Phase 2/3 randomized, controlled, dose-escalating study will be conducted at approximately 50 sites around the world and aims to enroll individuals of all ages with SMA.

Dr. Mishra[/caption]
Santosh K. Mishra M.Tech., PhD
Assistant Professor of Neuroscience
Department of Molecular Biomedical Sciences
NC State Veterinary Medicine
Raleigh, NC 2760
MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by atopic dermatitis?
Response: Chronic allergic itch is a worldwide problem that leads to substantial health expenses,but what causes this universal urge to scratch remains elusive in chronic allergic itch. Atopic dermatitis is a common allergic skin disease that often associated with extremely itchy and inflamed skin.
In our study, we showed, for the first time, a molecular pathway that is involved in chronic allergic itch as we identified an endogenous mediator (periostin) and a new role for its sensory neuron receptor, the integrin αVβ3, which drives the excitability and transmission of itch signal to the spinal cord.

The building of the filtered eye mask prototype.[/caption]

Dr. Hollingsworth[/caption]
Alex Hollingsworth PhD
Assistant Professor
O'Neill School of Public and Environmental Affairs
Indiana University
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: I've been working with Coady Wing and Ashley Bradford on a few different studies of the effects of recreational marijuana laws on drug and alcohol use.
Soon after EVALI became a major issue, the prevailing theory from the CDC and others was that EVALI was caused by the use of vitamin E acetate in illegal THC vaping products.
Our group read about this and we thought about some of the things that often happen in black markets for illegal drugs. For instance, during the alcohol prohibition era, bootleg alcohol producers often made and sold alcohol products that were not that safe to drink. In more recent years, there are cases where black market sellers of illegal drugs like heroin try to increase profit margins by adding other substances, which can be harmful.
We thought that maybe something like that could be happening in EVALI. Perhaps people in states where recreational marijuana is legal tended to purchase marijuana products from the legal market and the legal market was not selling any marijuana vaping products that included vitamin E acetate.
Barbara Chaiyachati, MD PhD
SafePlace: The Center for Child Protection and Health
Division of General Pediatrics
The Children’s Hospital of Philadelphia,
Center for Pediatric Clinical Effectiveness
University of Pennsylvania
Philadelphia, Pennsylvania
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Children in foster care have high rates of medical problems including chronic diseases. There is less known about the differences in mortality for children in foster care.
Looking at national data from 2003 to 2016, this study finds that children (ages 1 to 18) in foster care have higher mortality compared to children in the general population and that the difference in mortality has increased over time.

Vasily Giannakeas[/caption]
Vasily Giannakeas, MPH
Epidemiologist/ Dedicated ICES Analyst
Women's College Hospital
Toronto, Ontario, Canada
MedicalResearch.com: What is the background for this study?
Response: As some health care systems approach collapse, a pressing need exists for tools modeling the capacity of acute and critical care systems during the COVID-19 pandemic.
We developed an online tool to estimate the maximum number of COVID-19 cases that could be managed per day within the catchment area served by a health care system, given acute and critical care resource availability.
The COVID-19 Acute and Intensive Care Resource Tool (CAIC-RT) is open access and available at


