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Medicine

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Health, Epidemiology

New Study Design Holds Promise for Drug Safety Research

As the pace of drug approvals accelerates and the U.S. Food and Drug Administration (FDA) faces potential budget cuts, a new research design from Perelman School of Medicine scientists offers a new way to successfully assess safety of newly approved drugs, as well as drugs that have been on the market for a long time and have had a marked rise in their use. The study, published in the July issue of the journal Epidemiology, offers benefits over typically used randomized clinical trials, as such studies are often too small to identify rare side effects or may be performed in a group of patients who do not take other types of medications or have other conditions that could skew the drug's effect in a broader group following approval. Also first-in-class drugs may not have an applicable comparator drug, and traditional follow-up studies may give inaccurate results if those who take a new drug are different from those who took the comparator drug.

Science

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Neurodegeneration, Neurodegenative Disease, ALS, Alzheimer's, liquid-liquid phase transition

Gel-Like Drops of Protein and RNA Make a Splash in Neurodegenerative Diseases

At a meeting in Leuven, Belgium, a coherent picture began to emerge for how fluid pockets of proteins and RNAs contribute to health and disease.

Medicine

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Alzheimer's Disease, Dementia, Metabolic Conditions

Family History of Alzheimer’s May Alter Metabolic Gene That Increases Risk for Disease

A new Iowa State University study may have identified the link that explains years of conflicting research over a mitochondrial gene and the risk for Alzheimer’s disease.

Medicine

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Bone Health, epigenetic changes, Dna Methylation, Children, Pediatrc

Early Epigenetic Switches Associated with Childhood Bone Health

The health of children’s bones could be determined before they are born, a new University of Southampton study has shown.

Medicine

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Practical Clinical Trials Can Help Find Alternatives to Opioids

Pressures on primary care doctors to move away from opioid pain management are increasing, but practitioners need practical, evidence-based information on how to employ multidisciplinary pain care successfully in everyday clinical practice. A senior investigator for Kaiser Permanente, speaking at the American Pain Society Annual Scientific Conference, believes wider use of practical clinical trials and more emphasis on patient self-management are key solutions for achieving wider use of multidisciplinary pain care to improve patient function and help lower use and misuse of opioids.

Medicine

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citrin, citrullinemia, Erez, cancer metabolism

Rare Genetic Defect May Lead to Cancer Drug

Dr. Ayelet Erez, a doctor and geneticist at the Weizmann Institute of Science, has found that while a lack of the protein citrin slows children’s growth, blocking it in cancer slows tumor growth

Medicine

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Blood Test, Healthcare, Infectious Disease, Healthcare Costs, drug-resistant infections, Public Health

New Blood Test Technology Reduces False Readings, Saves Costs, and Improves Care - Live Virtual Press Briefing with Researcher May 16

Research findings to be published about new blood test technology that will greatly reduce errors in labwork and improve care in public health and infectious disease. Press briefing scheduled for May 16, reserve press access to live virtual event now.

Medicine

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Peter Campochiaro, Gene Therapy, Injections, Therapeutic

New Gene Therapy for Vision Loss Proven Safe in Humans

injection_syringe_tech_iStock-658483836TW.jpg

In a small and preliminary clinical trial, Johns Hopkins researchers and their collaborators have shown that an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving the vision of people with wet age-related macular degeneration (AMD).

Medicine

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Opthalmalogy, Ageing, Health, University of Birmingham

University of Birmingham Develops Revolutionary Eye Drops to Treat Age-Related Blindness

Scientists at the University of Birmingham have developed a type of eye drop which could potentially revolutionise the treatment of one of the leading causes of blindness in the UK.

Medicine

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Autism, Metformin, type 2 diabetes drugs, Fragile X Syndrome, intellectual disabilities, University of Montreal, McGill University , University Of Edinburgh, mice, Genetic Diseases, Brain, Protein, Neurological Disease, Mental Retardation, FMR1 mutation, speech impaired, Anxiety Disorders, Seizures, Biochemistry, Nature Medicine, Clinical Trials, Medication, Ne

Diabetes Drug May Help Symptoms of Autism-Associated Condition

Metformin, the most widely used drug to treat type 2 diabetes, could potentially be used to treat symptoms of Fragile X syndrome, an inherited form of intellectual disability and a cause of some forms of autism.







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