Sentences with phrase «therapy trials on»

Scientists believe that is what happened during a 1999 French gene therapy trial on a group of 10 young children with X-SCID, an immune deficiency disorder known as boy - in - the - bubble syndrome.

Scientists believe that's what happened during a 1999 French gene therapy trial on a group of 10 infants with X-SCID, an immune deficiency disorder known as boy - in - the - bubble syndrome.

Not exact matches

On the logistics end, it is easier to manufacture certain stem - cell therapies, which will be key for human trials like the heart - muscle - regeneration program.

While it takes time for gene therapies to be tested, trialed, and possibly approved by the FDA, genetics startups like Spark, AveXis, Caribou Biosciences, Editas, and Audentes have been on the road a while.

These risks and uncertainties include: Gilead's ability to achieve its anticipated full year 2018 financial results; Gilead's ability to sustain growth in revenues for its antiviral and other programs; the risk that private and public payers may be reluctant to provide, or continue to provide, coverage or reimbursement for new products, including Vosevi, Yescarta, Epclusa, Harvoni, Genvoya, Odefsey, Descovy, Biktarvy and Vemlidy ®; austerity measures in European countries that may increase the amount of discount required on Gilead's products; an increase in discounts, chargebacks and rebates due to ongoing contracts and future negotiations with commercial and government payers; a larger than anticipated shift in payer mix to more highly discounted payer segments and geographic regions and decreases in treatment duration; availability of funding for state AIDS Drug Assistance Programs (ADAPs); continued fluctuations in ADAP purchases driven by federal and state grant cycles which may not mirror patient demand and may cause fluctuations in Gilead's earnings; market share and price erosion caused by the introduction of generic versions of Viread and Truvada, an uncertain global macroeconomic environment; and potential amendments to the Affordable Care Act or other government action that could have the effect of lowering prices or reducing the number of insured patients; the possibility of unfavorable results from clinical trials involving investigational compounds; Gilead's ability to initiate clinical trials in its currently anticipated timeframes; the levels of inventory held by wholesalers and retailers which may cause fluctuations in Gilead's earnings; Kite's ability to develop and commercialize cell therapies utilizing the zinc finger nuclease technology platform and realize the benefits of the Sangamo partnership; Gilead's ability to submit new drug applications for new product candidates in the timelines currently anticipated; Gilead's ability to receive regulatory approvals in a timely manner or at all, for new and current products, including Biktarvy; Gilead's ability to successfully commercialize its products, including Biktarvy; the risk that physicians and patients may not see advantages of these products over other therapies and may therefore be reluctant to prescribe the products; Gilead's ability to successfully develop its hematology / oncology and inflammation / respiratory programs; safety and efficacy data from clinical studies may not warrant further development of Gilead's product candidates, including GS - 9620 and Yescarta in combination with Pfizer's utomilumab; Gilead's ability to pay dividends or complete its share repurchase program due to changes in its stock price, corporate or other market conditions; fluctuations in the foreign exchange rate of the U.S. dollar that may cause an unfavorable foreign currency exchange impact on Gilead's future revenues and pre-tax earnings; and other risks identified from time to time in Gilead's reports filed with the U.S. Securities and Exchange Commission (the SEC).

«There's more emphasis now and there will be more emphasis because of the Affordable Care Act [on] offering mental health services in primary care settings,» suggests Curry, who is about to start a clinical trial looking at the effects of cognitive behavior therapy for adolescents with depression and substance abuse problems.

Written on October 30, 2013 at 12:20 pm Categories: Home Blogroll, Thirsties Therapy Help Tags: by Sonya, cloth diaper trial programs, cloth diapers

A randomized placebo - controlled trial of massage therapy on the immune system of preterm infants.

The researchers caution that the booster therapy used in their new study will not be available on the market or even for use in human trials anytime soon; it must await years of animal testing for safety and effectiveness first.

Six lipid - lowering trials randomized 4,449 patients, who previously had a heart attack or stroke or had extremely high baseline cholesterol levels and were on statin therapy, to receive either bococizumab (150 mg subcutaneously every 2 weeks) or placebo to determine the effects on LDL levels.

But sometimes, they also have the privilege of seeing a therapy they worked on go to clinical trials or even to patients.

Most clinical trials on beta blockers took place before reperfusion therapy became standard treatment following heart attacks.

Based on results of the current study described in a report online June 18 in the journal Cancer Cell, Johns Hopkins researchers say they are planning a phase I clinical trial to test the paclitaxel - fostamatinib combination therapy in patients with recurrent advanced ovarian cancer.

«Current therapies in clinical trials are focused on targeting genetic changes in tumors and helping to boost one's immune system to fight the cancer cells.

AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXStherapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXSTherapy Designation for the treatment based on preliminary clinical results from the trial of AVXS - 101.

The CO-STAR (Hepatitis C Patients on Opioid Substitution Therapy Antiviral Response) trial sought to evaluate the efficacy and safety of elbasvir - grazoprevir for injection drug users.

Human trials are even costlier, so for now, Sweeney says, IGF - 1 and myostatin gene therapies remain on the distant horizon.

This was caused by an erroneous and incomplete search by the manufacturer for studies on the comparator therapy; no search at all was conducted in trial registries.

Because the impact of autophagy on cancer seems to go both ways, cancer trials have tested therapies that enhance autophagy as well as drugs that block it.

«Our results indicate it is a promising agent for clinical trials, and a as an add - on to more conventional therapies, to prevent tumour recurrence, further disease progression and metastasis.»

For example, in the combined hormone therapy trial, treated 50 - to 59 - year - olds had five additional cases of heart disease and five more strokes per 10,000 women annually compared with the same - aged group on placebo.

He says results from phase II and III trials on these new therapies designed to stall the disease are slated to come out within the year.

Clinical trials for targeted therapies, selected on the basis of the genomic tests, are scheduled to begin in early 2007.

Along with the focus on prevention, the WHI hormone therapy trials were largely studies of older women — in their 60s and 70s.

Although no clinical trials involving therapies derived from iPS cells are on the books, researchers are currently testing drugs on them.

«I think [there's] a lot more of an understanding about what's feasible, what's safe, what's ethical,» says Anna David, who studies fetal therapy at University College London and is collaborating with Semler on the OI trial.

Before moving on to human trials, they will need to study all instances of «off - target» effects: Years before Crispr, the viruses employed to deliver DNA in gene therapy trials occasionally damaged the whole system, causing cancer.

Antiretroviral therapy (ART) for HIV infection provides lasting protection against the sexual transmission of the virus from infected men and women to their HIV - uninfected sexual partners, investigators from the HIV Prevention Trials Network (HPTN) reported today at the 8th International AIDS Society (IAS) Conference on HIV Pathogenesis, Treatment and Prevention in Vancouver, Canada.

A large trial showing that men with newly diagnosed, advanced prostate cancer lived ten months longer, on average, when they received docetaxel chemotherapy along with standard hormone therapy

Sean Morrison, Professor and Director of the Children's Medical Center Research Institute at UT Southwestern Medical Center in the United States, who is not an author on the paper, added: «Patients are ultimately not helped by therapies that are not based on sound science and that are not tested in systematic clinical trials.

He added: «The most difficult decision was whether or not to have a period of initial triple therapy after stent implantation, given the findings of the WOEST trial which implied an adverse effect of triple therapy on bleeding and death.

Some on the panel suggested they were particularly sensitive about such concerns given that it was at UPenn in 1999 that a young man, Jessie Gelsinger, died in a gene therapy trial, setting the field back for years.

ACT, which has almost gone under several times in recent years, may now be on the verge of conducting the nation's first trial of an embryonic stem cell therapy, says Chief Scientific Officer Robert Lanza.

The placebo - controlled, double - blinded trial examined the effect of testosterone therapy on sexual function in a group of 470 men.

«We are excited that our translational research team of clinicians and basic researchers have come together to present these novel findings and hope that future studies and trials based on this work can move forward to bring targeted therapies for TED,» commented Dr. Phipps.

The lab is also working with the Breast Oncology Program at UCSF to make this data part of an adaptive clinical trial called I - SPY, which lets researchers identify the most effective therapies based on patient molecular profiling, and is collaborating with members of the UCSF Institute for Computational Health Sciences (ICHS) to put these and other public data into a centralized database that clinicians can access through an app to help make the most appropriate treatment decisions.

A panel of top science administrators and stem cell scientists met on Friday with Davide Vannoni, the president of an Italian foundation providing highly controversial stem cell treatments, in a first effort to hash out the details of a government - sponsored clinical trial of the therapy.

In future, Bandyopadhyay says, better understanding how chemotherapy agents impact specific biological pathways should allow drug trials to focus on patients who are more likely to respond to the drugs being tested and enable clinicians to identify targeted or combination therapies for patients with a genetic predisposition to resistance.

Commenting on the trial, Dr Alice Shaw, director of thoracic oncology at the Massachusetts General Hospital Cancer Centre in Boston, US, said: «This is the first randomised study to examine how a second generation ALK inhibitor compares to standard second line chemotherapy in ALK positive patients who failed the standard first line therapy, which currently is crizotinib.»

The results of the Endovascular Therapy Following Imaging Evaluation for the Ischemic Stroke (DEFUSE 3) trial, presented at the International Stroke Conference 2018 in Los Angeles and published on Jan. 24 in the New England Journal of Medicine, demonstrated that physically removing brain clots up to 16 hours after symptom onset in selected patients led to improved outcomes compared to standard medical tTherapy Following Imaging Evaluation for the Ischemic Stroke (DEFUSE 3) trial, presented at the International Stroke Conference 2018 in Los Angeles and published on Jan. 24 in the New England Journal of Medicine, demonstrated that physically removing brain clots up to 16 hours after symptom onset in selected patients led to improved outcomes compared to standard medical therapytherapy.

«First trial of gene therapy for cystic fibrosis to show beneficial effect on lung function.»

«We now suggest all patients with HIV who are started on antiviral treatment be started on combination therapy, and not just given a single agent,» says Brian Gazzard, principal investigator in the British arm of the trial.

The FDA's medical reviewer's recommendation for approval says, in part, that more trials before approval would «significantly delay effective therapy,» which not only reduces body weight but exerts favorable effects on blood pressure and myocardial oxygen for «patients with a serious disease condition with few treatment options.»

«The recent niacin clinical trials offer important new evidence that raising «good» cholesterol (HDL) levels on top of statin therapy does not have the positive outcome that had been hoped for,» said Neil Stone, M.D., the Robert Bonow MD Professor in Cardiology at Feinberg and a cardiologist at Northwestern Memorial Hospital.

Trials of combination therapy are supported by the American Thyroid Association, but clinical studies are mixed on whether this approach works, Bianco said.

Findings from the CONFIRM - HF (Ferric CarboxymaltOse evaluatioN on perFormance in patients with IRon deficiency in coMbination with chronic Heart Failure) trial, presented as an ESC Hot Line and published in the European Heart Journal, point to a simple and safe therapy for a frustratingly common problem, said investigator Piotr Ponikowski, MD, PhD, from Medical University in Wroclaw, Poland.

A new study by The HIV Prevention Trials Network (HPTN) shows that financial incentives did not have an overall effect on motivating HIV - positive patients to take their HIV antiretroviral therapy (ART) medication regularly and maintain control of their HIV.

Studies have shown it is well tolerated in HIV - positive patients on antiretroviral therapy, and several clinical trials are under way.

Jonathan Appleby, GSK project leader for the ADA - SCID trial and head of cell and gene therapy at the rare diseases unit, says there are many differences between working on this kind of trial and working with one for a common disease.

And it called for a nationwide moratorium on new clinical trials for stem - cell therapies, adding that patients in existing clinical trials should not be charged.