Treatment

Three-Drug Therapy for Most Common Genetic Cause of Cystic Fibrosis Found Safe and Effective in 6-11-Year-Olds

An international, open-label Phase 3 study, co-led by Susanna McColley, MD, from Ann & Robert H. Lurie Children’s Hospital of Chicago, found that a regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of cystic fibrosis was safe and effective in 6-11-year-olds with at least one copy of F508del mutation in the CFTR gene, which is estimated to represent almost 90 percent of the cystic fibrosis population in the United States.

Peginterferon-lambda shows strong antiviral action to accelerate clearance of COVID-19

A clinical study led by Dr. Jordan Feld, a liver specialist at Toronto Centre for Liver Disease, University Health Network (UHN), showed an experimental antiviral drug can significantly speed up recovery for COVID-19 outpatients – patients who do not need to be hospitalized. This could become an important intervention to treat infected patients and help curb community spread, while COVID-19 vaccines are rolled out this year.

Smartphone Use Offers Tool to Treat MS, Other Diseases

Monitoring how patients with multiple sclerosis or other degenerative diseases use their smartphones could provide valuable information to help get them better treatment. In the journal Chaos, researchers used an app to record the keystroke dynamics of a control group and those of subjects in various stages of MS treatment. In doing so, they observed changes in the way people with MS typed that were not seen in subjects who did not have the disease.

FDA Approves First At-Home Saliva Collection Test for Coronavirus

Rutgers’ RUCDR Infinite Biologics received an amended emergency use authorization from the FDA late Thursday for the first SARS-CoV-2 coronavirus test that will allow people to collect their own saliva at home and send to a lab for results. The decision follows the FDA’s recent emergency approval to RUCDR Infinite Biologics for the first saliva-based test, which involves health care workers collecting saliva from individuals at testing sites.

Researchers use live virus to identify 30 existing drugs that could treat COVID-19

Scientists at Sanford Burnham Prebys Medical Discovery Institute, the University of Hong Kong, Scripps Research, UC San Diego School of Medicine, the Icahn School of Medicine at Mount Sinai and UCLA have identified 30 existing drugs that stop the replication of SARS-CoV-2, the virus that causes COVID-19. The study was placed on bioRxiv (pronounced “bio-Archive”), an open-access distribution service for preprints of life science research.

Rutgers Launches Genetic Testing Service for New Coronavirus

Rutgers’ RUCDR Infinite Biologics has launched a test for the SARS-CoV-2 coronavirus and is using its automation experience and infrastructure to test as many as tens of thousands of samples daily. RUCDR has also submitted an emergency use authorization request for a saliva collection method that will allow for broader population screening.

Five language outcome measures evaluated for intellectual disabilities studies

Expressive language sampling yielded five language-related outcome measures that may be useful for treatment studies in intellectual disabilities, especially fragile X syndrome. The measures were generally valid and reliable across the range of ages, IQs and autism symptom severity of participants. According to the study, led by UC Davis researchers and funded by NIH, the measures are also functional in supporting treatments that can improve language, providing far reaching benefits for individuals with intellectual disabilities.

COVID-19 Appears Less Severe in Children, Says Review in Pediatric Infectious Disease Journal

As outbreaks of COVID-19 disease caused by the novel severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) continue worldwide, there’s reassuring evidence that children have fewer symptoms and less severe disease. That’s among the insights provided by an expert review in The Pediatric Infectious Disease Journal, the official journal of The European Society for Paediatric Infectious Diseases. The journal is published in the Lippincott portfolio by Wolters Kluwer.

COVID-19 Appears Less Severe in Children, Says Review in Pediatric Infectious Disease Journal

As outbreaks of COVID-19 disease caused by the novel severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) continue worldwide, there’s reassuring evidence that children have fewer symptoms and less severe disease. That’s among the insights provided by an expert review in The Pediatric Infectious Disease Journal, the official journal of The European Society for Paediatric Infectious Diseases. The journal is published in the Lippincott portfolio by Wolters Kluwer.

Investigational Drug for People with Treatment-Resistant Epilepsy

Imagine not being able to drive, shower alone or even work because you are never quite sure when the next seizure will leave you incapacitated. Hope may be on the horizon for epilepsy patients who have had limited success with seizure drugs. In a study, led by a Johns Hopkins lead investigator, of 437 patients across 107 institutions in 16 countries, researchers found that the investigational drug cenobamate reduced seizures 55% on the two highest doses of this medication that were tested over the entire treatment period.

Better Biosensor Technology Created for Stem Cells

A Rutgers-led team has created better biosensor technology that may help lead to safe stem cell therapies for treating Alzheimer’s and Parkinson’s diseases and other neurological disorders. The technology, which features a unique graphene and gold-based platform and high-tech imaging, monitors the fate of stem cells by detecting genetic material (RNA) involved in turning such cells into brain cells (neurons), according to a study in the journal Nano Letters.