Category Archives: Genetic Therapy

Shock Therapy: New Twist on an Old Treatment for Depression – Research Summary

BACKGROUND: Depression is a medical illness that involves the brain. Continue reading

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Scientists Find Genetic Difference In Highly-Sensitive People

LOS ANGELES (CBS) Do people say you are highly sensitive whether it be physically or emotionally? While it can feel like a burden, it can also mean a greater capacity for work, love and compassion. I knew that I was more sensitive, that I took things deeper and harder, Ane Axford said Continue reading

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Genetic test can accurately predict spread of eye cancer

Researchers at Washington University School of Medicine in St. Continue reading

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Genetic Test Identifies Eye Cancer Tumors Likely to Spread

Newswise Researchers at Washington University School of Medicine in St. Louis have developed a genetic test that can accurately predict whether the most common form of eye cancer will spread to other parts of the body, particularly the liver. In 459 patients with ocular melanoma at 12 centers in the United States and Canada, the researchers found the test could successfully classify tumors more than 97 percent of the time. Continue reading

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Lung cancer molecular subtypes correlate with genetic alterations, patient's response to therapy

Published on May 14, 2012 at 12:51 AM Cancer therapies targeting specific molecular subtypes of the disease allow physicians to tailor treatment to a patient’s individual molecular profile. But scientists are finding that in many types of cancer the molecular subtypes are more varied than previously thought and contain further genetic alterations that can affect a patient’s response to therapy. A UNC-led team of scientists has shown for the first time that lung cancer molecular subtypes correlate with distinct genetic alterations and with patient response to therapy Continue reading

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Diagnosing a Rare, Genetic Disease

by Gary Emmett, M.D. Five years ago, a one-year-old boy named Connor was brought into my office for a second opinion. He had gone to a fine pediatrician on the Main Line who knew that something was very wrong, but did not know exactly what. Continue reading

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Molecular subtypes and genetic alterations may determine response to lung cancer therapy

Public release date: 11-May-2012 [ | E-mail | Share ] Contact: Dianne G. Shaw dgs@med.unc.edu 919-966-7834 University of North Carolina School of Medicine Cancer therapies targeting specific molecular subtypes of the disease allow physicians to tailor treatment to a patient’s individual molecular profile. Continue reading

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Genetic predictor of breast cancer response to chemotherapy

Public release date: 10-May-2012 [ | E-mail | Share ] Contact: Dr. Hilary Glover hilary.glover@biomedcentral.com 44-020-319-22370 BioMed Central Chemotherapy is a major first line defense against breast cancer. Continue reading

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Vertex: Two-Drug Cystic Fibrosis Therapy Improves Lung Function

Updated with stock price, analyst reaction. CAMBRIDGE, Mass. (TheStreet) — Vertex Pharmaceuticals(VRTX) released preliminary data Monday showing two drugs — the experimental VX-809 and currently marketed Kalydeco — significantly improved lung function in patients with the most common genetic mutation causing cystic fibrosis. Continue reading

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DiaGenic ASA: DiaGenic Reports Completion of Data Collection and Genetic Analyses in a Unique Study on Familial …

OSLO, Norway–(BUSINESS WIRE)– Regulatory News: DiaGenic ASA (OSE:DIAG.OL – News): DiaGenic today reports on the finalization of data collection and database lock of a blinded study in a Norwegian cohort of 80 patients with familial Parkinsons disease (PD). Continue reading

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Not all tumor cells are equal: Stanford study reveals huge genetic diversity in cells shed by tumors

Public release date: 7-May-2012 [ | E-mail | Share ] Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center STANFORD, Calif. The cells that slough off from a cancerous tumor into the bloodstream are a genetically diverse bunch, Stanford University School of Medicine researchers have found Continue reading

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New Genetic Discoveries and Treatment for Hepatitis C [Viewpoint]

Michael Pacanowski, PharmD, MPH; Shashi Amur, PhD; Issam Zineh, PharmD, MPH Author Affiliations: Genomics Group, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland. Treatment of chronic hepatitis C (CHC) is a prototype for personalized medicine. Combination therapy with peginterferon alfa plus ribavirin was the standard of care for more than a decade Continue reading

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Lewis speaks on gene therapy at Lexington Community Education event

In a recent talk sponsored by Lexington Community Education, author and genetics expert Ricki Lewis wrapped a lecture on human genetics and gene therapy in the genuinely moving stories of children whose lives were altered — some for good, some … Continue reading

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Hot flashes more likely for certain smokers

THURSDAY, May 3 (HealthDay News) — Women smokers with certain gene variants are at increased risk for menopausal hot flashes compared to smokers without these genetic differences, a new study says. An analysis of data from nearly 300 late reproductive-age women who were followed for 11 years showed that smokers with specific variations (single nucleotide polymorphisms) in genes that affect metabolism are more likely to have hot flashes than smokers without these gene variants. Continue reading

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Life-saving enzyme eludes genetic disorder victim

Jharkhand Muslim Majlis Musawar, which had sponsored Sadaf's trip to Delhi, has sought a fresh appointment with the Ranchi MP to press for Sadaf's free treatment. Continue reading

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Researchers Discover First Gene Linked to Missing Spleen in Newborns

New Discovery of a Genetic Mutation in Congenital Asplenia May Lead to Genetic Prenatal Screening in Patients with the Rare, But Deadly, Disorder Newswise NEW YORK (May 3, 2012) — Researchers at Weill Cornell Medical College and Rockefeller University have identified the first gene to be linked to a rare condition in which babies are born without a spleen, putting those children at risk of dying from infections they cannot defend themselves against. Continue reading

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Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease

NOVATO, Calif., May 1, 2012 /PRNewswire/ –Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced results from a first-in-human, multi-center, Phase 1 study of UX001 in patients with hereditary inclusion body myopathy (HIBM) showing that UX001 was well-tolerated with an expected extended release profile on absorption after oral administration. UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency Continue reading

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FDA approves new orphan drug to treat a form of Gaucher disease

FDA NEWS RELEASE For Immediate Release: May 1, 2012 Media Inquiries: Stephanie Yao, 301-796-0394, stephanie.yao@fda.hhs.gov Consumer Inquiries: 888-INFO-FDA FDA approves new orphan drug to treat a form of Gaucher disease The U.S. Food and Drug Administration today approved Elelyso (taliglucerase alfa) for long-term enzyme replacement therapy to treat a form of Gaucher disease, a rare genetic disorder. Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. Continue reading

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Elelyso Approved for Gaucher Disease

TUESDAY, May 1 (HealthDay News) — Elelyso (taliglucerase alfa) has been approved by the U.S. Food and Drug Administration as a long-term enzyme replacement therapy for people with a rare genetic disorder called type 1 Gaucher disease. Continue reading

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