A00-281 exam Dumps Source : Clinical Trials Programming Using SAS 9 Accelerated Version
Test Code : A00-281
Test appellation : Clinical Trials Programming Using SAS 9 Accelerated Version
Vendor appellation : SASInstitute
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The branch of Biostatistics within the school of Public health and health Professions these days launched a brand unique analysis software for undergraduates perquisite through the 2018-19 wintry weather smash.
The Biostatistics Epidemiology and research Design (BERD) Undergraduate wintry weather Institute for Biostatistics became designed to address the widening cavity between the demand for qualified biostatisticians and the available give via pupil exposure to the self-discipline. This application provided 13 undergraduates with didactic lecture latitude instruction in simple biostatistical and epidemiological methodology, in addition to mentorship from individual BERD faculty, to befriend college students exist mindful the way to effectively result the methodology in an actual clinical and translational research surroundings.
The inaugural BERD wintry weather Institute kicked off in December with every week of on-site classroom and guideline before the college students departed for the holidays. From Dec. 22 via Jan. 22, members worked remotely on analysis initiatives beneath school mentorship. perquisite through the path of the institute, members additionally discovered statistical programming the usage of R statistical application; explored graduate and career alternatives, and took piece in networking activities; visited the UB scientific and Translational analysis heart on the Buffalo Niagara clinical Campus; and won firsthand insight from alumni in the box. When the college students returned to campus in January, the institute culminated with a poster presentation.
“for almost outright of the college students, this is their first experience in analysis. a lot of them believe now expressed hobby in pursuing extra training and a profession in biostatistics after completion of their undergraduate reviews,” says Gregory E. Wilding, chair of the department of Biostatistics.
“we're so lucky to proffer them this occasion to participate and expose them to percentages for their future.”
The program is supported by way of the UB scientific and Translational Science Institute (CTSI) through a vouchsafe from the countrywide heart for Advancing Translational Sciences of the countrywide Institutes of health (UL1TR001412) and by way of the UB department of Biostatistics. Participation in the program is offered at no cost, together with outright vital tutorial substances, nutrients and a treasure trove of talents to select with them.
“Biostatisticians play a key role in the design and behavior of rigorous medical analysis stories,” says Timothy F. Murphy, director of the CTSI, SUNY individual Professor of medication and senior associate dean for scientific and translational analysis within the Jacobs college of drugs and Biomedical Sciences at UB. “This exciting program aligns well with an Important intention of the CTSI to drill the subsequent generation of scientific and translational researchers. It changed into brilliant to peer the keenness of these talents future biostatisticians.”
besides Wilding, SPHHP faculty assisting with the institute included Dietrich Kuhlmann, Jeffrey Miecznikowski, Guan Yu, Jiwei Zhao, Austin Miller and Michael LaMonte.
skill degree: Intermediate status: lively
low-priced: $one hundred eighty (shortest song)
abstract:For programmers and statisticians who expend SAS software to investigate clinical trials records to provide reviews for submission to regulatory authorities. contour certain to believe journey in the medical trials manner, getting access to, managing, and remodeling scientific trials facts, statistical tactics and macro programming, reporting medical trials outcomes, and validating scientific tribulation statistics reporting.
preliminary requirements:There are two the perquisite way to merit this credential:
1) flow the scientific Trials Programming the usage of SAS 9 examination ($180). The examination consists of 99 questions and has a three hour closing date. A passing ranking of 70% is required.
2) in case you already cling the SAS foundation Programmer credential, you could select the accelerated version of the scientific Trials Programming using SAS 9 examination ($180). This shorter exam does not iterate cloth coated on the bottom programming exam.The accelerated examination carries seventy one questions and has a 2 hour deadline. A passing rating of 70% is required.
practicing is attainable but not required.
continuing necessities:The SAS international Certification application now considerations versioned credentials that don't expire. The version is indicated in the credential identify - for instance, SAS licensed foundation Programmer for SAS 9. prior to now, SAS issued non-versioned credentials and required recertification every three years, however with the addition of versioning to the credential names, recertification necessities believe been dropped.
See outright Sas Certifications
supplier's web page for this certification
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Skill Level: Intermediate Status: Active
Low Cost: $180 (shortest track)
Summary:For programmers and statisticians who expend SAS software to anatomize clinical trials data to bear reports for submission to regulatory authorities. You should believe experience in the clinical trials process, accessing, managing, and transforming clinical trials data, statistical procedures and macro programming, reporting clinical trials results, and validating clinical tribulation data reporting.
Initial Requirements:There are two ways to merit this credential:
1) Pass the Clinical Trials Programming Using SAS 9 exam ($180). The exam consists of 99 questions and has a 3 hour time limit. A passing score of 70% is required.
2) If you already hold the SAS foundation Programmer credential, you can select the accelerated version of the Clinical Trials Programming Using SAS 9 exam ($180). This shorter exam doesn't iterate material covered on the foundation programming exam.The accelerated exam consists of 71 questions and has a 2 hour time limit. A passing score of 70% is required.
Training is available but not required.
Continuing Requirements:The SAS Global Certification Program now issues versioned credentials that conclude not expire. The version is indicated in the credential appellation - for example, SAS Certified foundation Programmer for SAS 9. Previously, SAS issued non-versioned credentials and required recertification every 3 years, but with the addition of versioning to the credential names, recertification requirements were dropped.
See outright Sas Certifications
Vendor's page for this certification
Completed Enrollment of topic Cohort to back BLA Submission Through Accelerated Approval (AA) Pathway for Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
CHMP sentiment for Palynziq® (pegvaliase) Injection for Patients 16 and older with PKU Anticipated in 1Q 2019 and Potential Approval in EU in 2Q 2019
SAN RAFAEL, Calif., Jan. 7, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (BMRN), a company focused on innovative therapies to deal rare diseases, provided highlights to the investment community on its key milestones for 2019 at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco.
"With multiple development and regulatory milestones expected in 2019, BioMarin maintains stalwart pipeline momentum. They are looking forward to advancing multiple product candidates in parallel through the development process," said Jean-Jacques Bienaimé, BioMarin Chairman and CEO. "Our focus on the unmet needs in rare genetic diseases and demonstrating meaningful clinical benefits provides the foundation to journey rapidly through product development and commercialization."
During the company's presentation at the conference, Bienaimé provided information on the company's development program for valoctocogene roxaparvovec gene therapy for stern hemophilia A. BioMarin has completed enrollment of the initial cohort of patients in its phase 3 program intended to back a BLA submission through the accelerated approval pathway. A determination to submit a BLA through an accelerated approval pathway is tracking for the second half of 2019. If the company submits a BLA using the accelerated approval pathway, it will disclose additional information on the timing of its plans regarding the BLA submission. The complete phase 3 study is targeting enrollment of 130 patients by mid-year 2019.
Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the European Medicines Agency (EMA). Valoctocogene roxaparvovec has too been accepted for Priority Medicines (PRIME) scheme from the EMA. Additionally, the FDA has granted valoctocogene roxaparvovec Breakthrough Therapy designation.
BioMarin too provided an update on its clinical program for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common contour of disproportionate short stature in humans.
BioMarin expects top line results from the fully enrolled phase 3 study of vosoritide in children by year nigh 2019. The phase 2 study in infants and young children up to age 5 with achondroplasia study enrollment is on track, and in this early piece of the study, vosoritide is generally well-tolerated. observe "About Vosoritide phase 3 Study" and "About Vosoritide phase 2 Infant and young Children Study" below for more information on the studies.
In addition, Bienaimé famous during the presentation that the company is anticipating an sentiment from the Committee for Medicinal Products for Human expend (CHMP), the scientific committee of the European Medicines Agency (EMA) on Palynziq® (pegvaliase) Injection for the treatment of patients 16 and older with PKU in 1Q 2019. If the CHMP provides a positive sentiment in 1Q 2019, then in 2Q 2019, it is viable that the European Commission (EC) could provide marketing authorization in the European Union. In addition, the company provided 2019 global revenue guidance for Palynziq of between $70 and $100 million.
Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme approved in the United States in May 2018, is the first approved enzyme substitution therapy to target the underlying understanding of phenylketonuria (PKU) by helping the body to shatter down Phe. PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. Palynziq is BioMarin's second approved treatment for this staid condition. In March 2018, the European Medicines Agency accepted BioMarin's submission of a Marketing Authorization Application for Palynziq.
Gene Therapy Manufacturing
BioMarin has constructed one of the largest gene therapy manufacturing facilities of its kind, which is located in Novato, California. noble Manufacturing Practices (GMP) production of valoctocogene roxaparvovec has commenced supporting clinical development activities and will back any anticipated commercial demand. This facility is capable of supporting approximately 4,000 doses per year, and the production process was developed in accordance with International Conference on Harmonisation guidance for Pharmaceuticals for Human expend to facilitate worldwide registration with health authorities. Clinical supplies for valoctocogene roxaparvovec for Hemophilia A and BMN 307, a pre-clinical gene therapy for PKU, are produced in this facility.
About BioMarin and Disease Information
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with staid and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on such website is not incorporated by reference into this press release.
About Hemophilia A
Hemophilia A, too called factor VIII (FVIII) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Although it can exist passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a unique mutation that was not inherited. Approximately 1 in 10,000 people predominantly manly is born with Hemophilia A. People alive with the disease are not able to contour blood clots efficiently and are at risk for inordinate bleeding from modest injuries, potentially endangering their lives. People with stern hemophilia often bleed spontaneously into their muscles or joints. The yardstick of custody for the 49% of individuals with hemophilia A who are severely affected is a prophylactic regimen of Factor VIII infusions two to three times per week. Even with prophylactic regimens, many people noiseless experience spontaneous bleeding events that result in progressive and debilitating joint damage.
Valoctocogene Roxaparvovec phase 1/2 Study Safety
Overall, valoctocogene roxaparvovec has been well-tolerated by patients across outright doses. No patients developed inhibitors to Factor VIII and no patients withdrew from the study. The most common AEs across outright dose cohorts were alanine aminotransferase (ALT) height (11 patients, 73%); arthralgia, aspartate aminotransferase elevation, and headache (7 patients each, 47%); back twinge and fatigue (5 patients each, 33%). Two patients reported SAEs during the study. One patient was hospitalized for observation after developing Grade 2 pyrexia with myalgia and headache within 24 hours of receiving valoctocogene roxaparvovec. The event resolved within 48 hours following treatment with paracetamol, an over-the-counter treatment for twinge and fever. The event was assessed as related to valoctocogene roxaparvovec. The other AE was assessed as not related to the therapy, attributed to a planned knee surgery to deal hemophilic arthropathy, and Grade 1 in severity. No complications were reported.
Achondroplasia, the most common contour of disproportionate short stature in humans, is characterized by failure of confidential conversion of cartilage into bone, which results in disproportionate short stature. This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience staid health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, studies demonstrate increased mortality at every age.
More than 80% of children with achondroplasia believe parents of tolerable stature and believe the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. Vosoritide is being tested in children whose growth plates are noiseless "open," typically those under 18 years of age. This is approximately 25% of people with achondroplasia. In the U.S., Europe, Latin America and the Middle East, there is currently no licensed medicines for achondroplasia.
About Vosoritide phase 3 Study
The global phase 3 study is a randomized, placebo-controlled study of vosoritide in approximately 110 children with achondroplasia ages 5-14 for 52 weeks. The study will exist followed by a subsequent open-label extension. Children in this study will believe completed a minimum six-month baseline study to determine their respective baseline growth velocity prior to entering the phase 3 study. Vosoritide is being tested in children whose growth plates are noiseless open. This is approximately 25% of people with achondroplasia.
The primary endpoint of the study is the change in growth velocity from baseline over one year in children treated compared to placebo. The company too plans to augment the growth velocity data with assessments of proportionality and functionality.
About Vosoritide phase 2 Infant and young Children Study
The phase 2 vosoritide study is a randomized, placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia ages zero to less than 60 months for 52 weeks. The study will exist followed by a subsequent open-label extension. Children in this study will believe completed a minimum three-month baseline study to determine their respective baseline growth prior to entering the phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the upshot of vosoritide on height Z-scores, which is the number of yardstick deviations in relation to the connote height of age-matched, tolerable stature children. The company too plans to augment the height Z-score data with assessments including proportionality, functionality, attribute of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries.
Vosoritide, administered in over 28,000 injections, was generally well tolerated at outright doses. The majority of adverse events (AEs) were mild and no staid adverse events (SAEs) were reported as study drug-related. Across outright doses, injection site reactions and hypotension were the most common drug-related AEs. All injection site reaction events were transient. AEs of hypotension were mild, momentary and resolved without medical intervention, and the majority were asymptomatic and reported in context of routine blood pressure measurements. No unique safety findings were observed at the 30 µg/kg/day dose.
PKU, or PAH deficiency, is a genetic disorder affecting approximately 90,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in adequate quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including stern intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually outright individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can exist managed with a Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, the strict diet is difficult for most adult patients to adhere to the extent needed for achieving adequate control of blood Phe levels.
To learn more about PKU and PAH deficiency, please visit www.PKU.com. Information on such website is not incorporated by reference into this press release.
Palynziq substitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with the PEGylated version of the enzyme phenylalanine ammonia lyase to shatter down Phe. Palynziq is administered using a dosing regimen designed to facilitate tolerability; Palynziq's safety profile consists primarily of immune-mediated responses, including anaphylaxis, for which robust risk management measures efficient in clinical trials are in place.
The dosing and administration of Palynziq follows an induction, titration, and maintenance paradigm. Treatment is individualized to the lowest efficient and tolerated dosage. Prescribers may reckon increasing to a maximum of 40 mg once daily in patients who believe not achieved a response with 20 mg once daily for at least 24 weeks. Prescribers are instructed to discontinue treatment in patients who believe not responded after 16 weeks of continuous treatment with the maximum dosage of 40 mg once daily. Periodic blood Phe monitoring is recommended, and patients should exist counseled on how to adjust their dietary intake, as needed, based on blood Phe concentrations.
Palynziq (pegvaliase-pqpz) Injection is a phenylalanine-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult patients with PKUwho believe uncontrolled blood phenylalanine concentrations greater than 600 µmol/L on existing management.
Important Safety Information
BOXED WARNING: RISK OF ANAPHYLAXIS
WARNINGS AND PRECAUTIONS
Other hypersensitivity reactions
Blood Phenylalanine Monitoring and Diet
Effect of PALYNZIQ on other PEGylated products
USE IN SPECIFIC POPULATIONS
Pregnancy and Lactation
You are encouraged to report side effects to report suspected adverse events to BioMarin at 1-877-695-8826 and the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please observe replete Prescribing Information, including Boxed Warning, at PALYNZIQ.com/hcp, which will exist available in 24 hours.
Forward Looking Statement
This press release contains forward-looking statements about the industry prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about its development programs and regulatory actions related to these programs, including the timing of (i) decisions by regulators, including the EMA's determination regarding BioMarin's MAA for Palynziq, (ii) BioMarin's global revenue guidance for Palyzniq in 2019 ( iii) BioMarin's preclinical studies and clinical studies and trials, (iv) completion of enrollment of those studies and trials, and (v) announcements of data from those studies and trials; the production capacity of BioMarin's gene therapy manufacturing facility; the continued clinical development and commercialization of BioMarin's commercial products and product candidates, including BioMarin's plans to complete the phase 3 study enrollment of valoctocogene roxaparvovec by mid-year 2019 and potentially rule in the second half of 2019 whether to submit a BLA for valoctocogene roxaparvovec for accelerated approval and the viable approval and commercialization of BioMarin's product candidates. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may vary materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of their product candidates, the continued clinical experiences of the patients in the current clinical studies; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities; their competence to successfully manufacture their product candidates for the preclinical and clinical trials; and those factors particular in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on contour 10-Q for the quarter ended September 30, 2018 as such factors may exist updated by any subsequent reports. Stockholders are urged not to plot undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any responsibility to update or alter any forward-looking statement, whether as a result of unique information, future events or otherwise.
BioMarin®, Palynziq®, and BioMarin are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates.
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc.
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