Sentences with phrase «efficacy of therapy for»

Not exact matches

Insurers like Anthem have refused to cover the exorbitantly pricey treatment (around $ 300,000) since the drug's label admits that Exondys hasn't proved its efficacy, making the therapy a roll of the dice for payers and patients alike.

The treatment was approved for the devastating rare disease Duchenne muscular dystrophy but created major divisions within the FDA because it simply hadn't demonstrated a whole lot of efficacy, meaning the pricey therapy is essentially being subjected to a real - world clinical test.

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).

However, for open - minded researchers, the efficacy of repartive therapy is still open to scientific investigation.

For the same reason, it is not easy to prove treatment efficacy by means of controlled, randomized studies, the «gold standard» for evaluating new therapiFor the same reason, it is not easy to prove treatment efficacy by means of controlled, randomized studies, the «gold standard» for evaluating new therapifor evaluating new therapies.

Countless studies have established the efficacy of cognitive behavioral therapy (CBT) for many manifestations of depression and anxiety.

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.

«Our findings point to the importance of physicians counseling their patients regarding the potential harms of smoking interfering with the efficacy of therapies and for increased risks of side effects.»

Dr. Rassool says that a clinical trial is planned to test whether low doses of a DNMT inhibitor, decitabine, and an investigational PARP inhibitor, talazoparib, can be safely combined and whether this therapy shows efficacy for AML patients, especially those who can not receive intensive chemotherapy, whose leukemia is resistant to treatment, or who have experienced a relapse after treatment.

In addition, the NFL should convene an independent committee of medical professionals, without ties to the NFL or any of its teams, to evaluate the safety and efficacy of the therapies, establishing a set of criteria for what is considered «safe» and «effective,» the authors said.

«We hope that this assessment of the efficacy of currently available migraine therapies helps patients and their physicians utilize treatments that are the most appropriate for them,» said Dr. Silberstein.

«These findings do not only contribute to new insights on the effects of anti-estrogen but also the potential of using miRNA for monitoring drug efficacy and for future RNA - based therapy developments.

«If we are able to show efficacy in non-human primates, we will begin work with the FDA to get approval for the use of this viral gene therapy in diabetic patients, both type 1 and type 2,» Gittes says.

Researchers in New Zealand studied 206 OA patients for two years to measure the efficacy of adding regular physical exercise, manual therapy, or a combination of both therapies to standard care, versus continuing usual medical care for OA alone.

Two new phase III clinical trials investigating the efficacy and safety of bitopertin, a glycine uptake inhibitor considered to be a promising new add - on therapy for treating negative symptoms in schizophrenia, failed to show a benefit of the drug over placebo.

If these preclinical studies are successful, the researchers plan to further develop their CAR T cell therapy and test its safety and efficacy for different types of metastatic cancer in upcoming clinical trials.

Fischer, in 2000, reported demonstrating the clinical efficacy of gene therapy for the first time, using blood stem cells to treat a fatal genetic disorder called X-linked severe combined immunodeficiency.

«The first year results from the CoreValve US Pivotal Trial support the safety and efficacy of this therapy in patients unsuitable for surgical aortic valve replacement,» said lead investigator Steven Yakubov, MD..

The results from our new study suggest that entinostat may enhance the anti-tumor efficacy of PD - 1 targeted therapy through MDSC targeting, potentially providing an effective combination treatment approach for patients with solid tumors, including lung and renal cell carcinoma.»

Scientists at the Universitat Autònoma de Barcelona (UAB) and the Catalan Institute for Nanoscience and Nanotechnology (ICN2) have developed a nanoencapsulation system with a liposome coating in order to increase the efficacy of bacteriophages in oral phage therapy.

For very rare diseases such as WAS, multicenter clinical trials are the only effective way of proving the safety and efficacy of gene therapy and having it rapidly approuved and made available to all patients.

French teams from CIC Biothérapie (AP - HP / Inserm), from pediatric hematology department of Necker Hospital for Children (AP - HP), led by Marina Cavazzana, Salima Hacein Bey Albina and Alain Fischer and from Genethon led by Anne Galy (Genethon / Inserm UMR - S951), and English teams from UCL Institute of Child Health and Great Ormond Street Hospital in London led by Adrian Thrasher and Bobby Gaspar demonstrated the efficacy of gene therapy treatment for Wiskott - Aldrich Syndrome (WAS).

Researchers explained that this difference may be due, in part, to a selection bias where healthier patients were treated with surgery, and they underscored the need for a controlled clinical trial with matched patients receiving each treatment first - line to compare the efficacy of these therapies.

The newly published PLOS ONE study assessed the long - term efficacy of the therapy and demonstrated that a single gene therapy treatment led to prolonged production of VNA in blood and protected the mice from subsequent exposures to C. botulinum toxin for up to several months.

«Efficacy of new gene therapy approach for toxin exposures shown in mouse study.»

As this is the first report on this PTH therapy for ischemic stroke for the demonstration of the efficacy and feasibility, PTH treatment was initiated at 1 hr after stroke followed by repeated administrations for 6 days.

A Randomized, Phase III Trial to Evaluate The Efficacy and Safety of MK - 3475 (Pembrolizumab) as Adjuvant Therapy for Triple Receptor - Negative Breast Cancer with ≥ 1 cm Residual Invasive Cancer or Positive Lymph Nodes (yPN +) After Neoadjuvant Chemotherapy

He has co-authored more than 100 scientific papers, abstracts, and letters examining the efficacy, safety and effects of drug therapies for cancer.

In line with the objectives of the International Rare Disease Research Consortium (IRDiRC), we aim to develop novel tools that will allow to make more accurate diagnoses, predict the disease course and the efficacy of available treatments, and help developing new and better therapies for rare kidney diseases.

Researchers are examining the efficacy of mindfulness - based cognitive therapy for improving emotional and behavioral problems following brain injury, such as aggression, anxiety, and depression.

ViaCyte's VC - 01 ™ product candidate, a first - in - class cell replacement therapy for the treatment of type 1 diabetes, is currently being evaluated in a Phase 1/2 trial called STEP ONE, or Safety, Tolerability, and Efficacy of VC - 01 Combination Product in Type One Diabetes.

Paris, France, April 3, 2018, 5.35 pm CET — GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA - PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced topline results from the REVERSE Phase III clinical trial evaluating the safety and efficacy of a single intravitreal injection of GS010 (rAAV2 / 2 - ND 4) in 37 subjects whose visual loss due to 11778 - ND4 Leber Hereditary Optic Neuropathy (LHON) commenced between 6 and 12 months prior to study treatment.

Ultimately, we use all this information to establish innovative culture techniques for generating unlimited neural stem cell sources for the derivation of specific types of clinically relevant neuronal or glial cells, for their use in disease modelling and drug discovery platforms, and towards refining their efficacy for future cell replacement - based therapy.

Clinical Applications and Bioprocessing Strategies Fundamentally, gene therapy should exceed the efficacy of existing therapeutic approaches and provide therapeutic strategies for unmet medical needs.

Scientists from Emulate and Roche form innovative partnership to use Organ - Chips for testing of efficacy and safety of new antibody therapeutics and combination therapies

Lysogene collaborates with University of Massachusetts Medical School (UMMS) for pre-clinical efficacy studies of innovative AAV - based gene therapy approaches in the GM1 gangliosidosis mouse model.

Despite the expansion of genetic profiling, there remains a large percentage of patients for whom no biomarkers of drug efficacy are detected or who become refractory to their targeted therapy or chemotherapy regimen.

Dr. Jain's nominators note that, in sum, his work has provided ``... unprecedented molecular, cellular, anatomical and functional insights into the vascular pathophysiology of tumors, for proposing the seminal hypothesis that antiangiogenic therapy can «normalize» the aberrant vasculature and microenvironment of tumors and thereby improve both delivery and efficacy of treatment of solid tumors.»

Though it is among the most common types of therapy, there is less evidence for its efficacy than there is for CBT.

Remarkable efficacy of thermal therapy for sjogren syndrome.

For the same reasons of safety and efficacy, we only use bioidentical hormones for estrogen replacement theraFor the same reasons of safety and efficacy, we only use bioidentical hormones for estrogen replacement therafor estrogen replacement therapy.

For a legitimate place where to get legal injectable HGH for sale online, you should contact a medical facility with the sole practice of hormone replacement therapy, where you will not only get safe and high - quality medicine, but you will also receive a full treatment program that includes the monitoring of treatment by qualified medical personnel (yet another means of ensuring both safety and efficacFor a legitimate place where to get legal injectable HGH for sale online, you should contact a medical facility with the sole practice of hormone replacement therapy, where you will not only get safe and high - quality medicine, but you will also receive a full treatment program that includes the monitoring of treatment by qualified medical personnel (yet another means of ensuring both safety and efficacfor sale online, you should contact a medical facility with the sole practice of hormone replacement therapy, where you will not only get safe and high - quality medicine, but you will also receive a full treatment program that includes the monitoring of treatment by qualified medical personnel (yet another means of ensuring both safety and efficacy).

The study measured the efficacy of massage therapy treatment by utilizing the Alcohol Withdrawal Scale (AWS), a tool for measuring physiological alcohol withdrawal symptoms such as one - minute radial pulse rate and respiration rate at the beginning and end of each 15 - minute massage or rest interval.

For more, see The effect of Saccharomyces boulardii on Candida albicans - infected intestinal cell lines Caco - 2 and Intestin - 407, and Efficacy and safety of the probiotic Saccharomyces boulardii for the prevention and therapy of gastrointestinal disordeFor more, see The effect of Saccharomyces boulardii on Candida albicans - infected intestinal cell lines Caco - 2 and Intestin - 407, and Efficacy and safety of the probiotic Saccharomyces boulardii for the prevention and therapy of gastrointestinal disordefor the prevention and therapy of gastrointestinal disorders.

Pelvic Floor Physical Therapy is effective for prolapses in stages 1 and 2 and its focus is the relief of pressure by means of hypopressive abdominal exercises, and the improvement of tone and endurance of the pelvic floor muscles to help maintain the position of the organs in the pelvis (1: Efficacy of pelvic floor muscle training and hypopressive exercises for treating pelvic organ prolapse in women: randomized controlled trial).

This one involves testing the efficacy of capsaicin, an active component of chili peppers, as a therapy for otherwise healthy dogs diagnosed with osteoarthritis in an elbow or limb joint.

If you scour veterinary literature, you won't find many double - blind clinical studies on therapy lasers; however, if you search PubMed for clinical evidence on the efficacy of laser therapy, you'll be inundated with thousands of articles on its various applications — ranging from its basic usages to the vastly more extreme and bizarre: things like treating diabetic ulcers, traumatic brain injuries, peripheral nervous system failures, mal - union fractures, and more.

Regularly scheduled laboratory work is important to monitor for efficacy of methimazole therapy and to identify any possible adverse effects.

WASHINGTON, D.C., June 3 — Help for the more than 12,000 children diagnosed with cancer each year in the United States, in the form of an innovative, four - legged therapy, is about to be rigorously studied to determine its efficacy.

Therefore, the purpose of this study is to examine the efficacy of stem cell therapy as a potential treatment for acute severe spinal cord injuries in dogs.