Emgality

NOTICE: This Consumer Medicine Information (CMI) is intended for persons living in Australia. EMGALITY® This medicine is subject to additional monitoring. This will allow quick identificationof new safety information. You can help by reporting any side effects you may get.You can report side effects to your doctor, or directly at www.tga.gov.au/reporting-problems.   Prefilled Pen (Autoinjector) […]

Cancer-fighting gene restrains ‘jumping genes’

About half of all tumors have mutations of the gene p53, normally responsible for warding off cancer. Now, UT Southwestern scientists have discovered a new role for p53 in its fight against tumors: preventing retrotransposons, or “jumping genes,” from hopping around the human genome. In cells with missing or mutated p53, the team found, retrotransposons […]

Research team identifies differences between benign and pathogenic gene variants

An international team of researchers led by Cleveland Clinic’s Lerner Research Institute has performed for the first time a wide-scale characterization of missense variants from 1,330 disease-associated genes. Published in Proceedings of the National Academy of Sciences, the study identifies features associated with pathogenic and benign variants that reveal the effects of the mutations at […]

Pfizer Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Investigational Gene Therapy PF-06939926

NEW YORK–(BUSINESS WIRE)–October 01, 2020 — Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA). PF-06939926 is currently being evaluated to determine the safety and efficacy of this gene therapy in […]

Twist on CRISPR gene editing treats adult-onset muscular dystrophy in mice

Myotonic dystrophy type I is the most common type of adult-onset muscular dystrophy. People with the condition inherit repeated DNA segments that lead to the toxic buildup of repetitive RNA, the messenger that carries a gene’s recipe to the cell’s protein-making machinery. As a result, people born with myotonic dystrophy experience progressive muscle wasting and […]

Study targets gene associated with Alzheimer’s disease

Researchers at the University of Alabama at Birmingham are on the track of a gene that might play a role in the development of Alzheimer’s disease. The research team is studying a gene called BIN1, which was first linked to Alzheimer’s disease in 2009. In a paper recently published online in eLife, the team shows […]

Bubble Boy! See Amy Schumer and Chris Fischer’s Family Album With Son Gene

The funny family! Amy Schumer and Chris Fischer have kept the laughs and love coming since tying the knot in February 2018. The comedian and chef wed in a surprise ceremony in February 2018 after dating for only a few months — and the ceremony included its share of jokes. “I’m so happy for you, that you found me,” […]

Gene targeting overcomes the resistance of brain cancer to therapy

New insight into a gene that controls energy production in cancer stem cells could help in the search for a more effective treatment for glioblastoma. A McGill-led study published in Nature Communications reveals that suppressing the OSMR gene can improve the effectiveness of radiation therapy. This approach, led by the laboratory of Arezu Jahani-Asl, Assistant […]

Restoring vision by gene therapy

Humans rely dominantly on their eyesight. Losing vision means inability to read, recognize faces or find objects. Macular degeneration is one of the major causes of visual impairment around the globe; close to 200 million people are affected. Photoreceptors in the retina are responsible for capturing the light coming from the environment. Diseased photoreceptors lose […]