Clinical Trials, Progression-Speed Differentiating Features, and Swiftness Rule of the Innovative Targets of First-In-Class Drugs
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AHFS Pharmacologic-Therapeutic Classification System
AHFS Pharmacologic-Therapeutic Classification System Abacavir 48:24 - Mucolytic Agents - 382638 8:18.08.20 - HIV Nucleoside and Nucleotide Reverse Acitretin 84:92 - Skin and Mucous Membrane Agents, Abaloparatide 68:24.08 - Parathyroid Agents - 317036 Aclidinium Abatacept 12:08.08 - Antimuscarinics/Antispasmodics - 313022 92:36 - Disease-modifying Antirheumatic Drugs - Acrivastine 92:20 - Immunomodulatory Agents - 306003 4:08 - Second Generation Antihistamines - 394040 Abciximab 48:04.08 - Second Generation Antihistamines - 394040 20:12.18 - Platelet-aggregation Inhibitors - 395014 Acyclovir Abemaciclib 8:18.32 - Nucleosides and Nucleotides - 381045 10:00 - Antineoplastic Agents - 317058 84:04.06 - Antivirals - 381036 Abiraterone Adalimumab; -adaz 10:00 - Antineoplastic Agents - 311027 92:36 - Disease-modifying Antirheumatic Drugs - AbobotulinumtoxinA 56:92 - GI Drugs, Miscellaneous - 302046 92:20 - Immunomodulatory Agents - 302046 92:92 - Other Miscellaneous Therapeutic Agents - 12:20.92 - Skeletal Muscle Relaxants, Miscellaneous - Adapalene 84:92 - Skin and Mucous Membrane Agents, Acalabrutinib 10:00 - Antineoplastic Agents - 317059 Adefovir Acamprosate 8:18.32 - Nucleosides and Nucleotides - 302036 28:92 - Central Nervous System Agents, Adenosine 24:04.04.24 - Class IV Antiarrhythmics - 304010 Acarbose Adenovirus Vaccine Live Oral 68:20.02 - alpha-Glucosidase Inhibitors - 396015 80:12 - Vaccines - 315016 Acebutolol Ado-Trastuzumab 24:24 - beta-Adrenergic Blocking Agents - 387003 10:00 - Antineoplastic Agents - 313041 12:16.08.08 - Selective -
Comparing the Effects of Combined Oral Contraceptives Containing Progestins with Low Androgenic and Antiandrogenic Activities on the Hypothalamic-Pituitary-Gonadal Axis In
JMIR RESEARCH PROTOCOLS Amiri et al Review Comparing the Effects of Combined Oral Contraceptives Containing Progestins With Low Androgenic and Antiandrogenic Activities on the Hypothalamic-Pituitary-Gonadal Axis in Patients With Polycystic Ovary Syndrome: Systematic Review and Meta-Analysis Mina Amiri1,2, PhD, Postdoc; Fahimeh Ramezani Tehrani2, MD; Fatemeh Nahidi3, PhD; Ali Kabir4, MD, MPH, PhD; Fereidoun Azizi5, MD 1Students Research Committee, School of Nursing and Midwifery, Department of Midwifery and Reproductive Health, Shahid Beheshti University of Medical Sciences, Tehran, Islamic Republic Of Iran 2Reproductive Endocrinology Research Center, Research Institute for Endocrine Sciences, Shahid Beheshti University of Medical Sciences, Tehran, Islamic Republic Of Iran 3School of Nursing and Midwifery, Department of Midwifery and Reproductive Health, Shahid Beheshti University of Medical Sciences, Tehran, Islamic Republic Of Iran 4Minimally Invasive Surgery Research Center, Iran University of Medical Sciences, Tehran, Islamic Republic Of Iran 5Endocrine Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Islamic Republic Of Iran Corresponding Author: Fahimeh Ramezani Tehrani, MD Reproductive Endocrinology Research Center Research Institute for Endocrine Sciences Shahid Beheshti University of Medical Sciences 24 Parvaneh Yaman Street, Velenjak, PO Box 19395-4763 Tehran, 1985717413 Islamic Republic Of Iran Phone: 98 21 22432500 Email: [email protected] Abstract Background: Different products of combined oral contraceptives (COCs) can improve clinical and biochemical findings in patients with polycystic ovary syndrome (PCOS) through suppression of the hypothalamic-pituitary-gonadal (HPG) axis. Objective: This systematic review and meta-analysis aimed to compare the effects of COCs containing progestins with low androgenic and antiandrogenic activities on the HPG axis in patients with PCOS. -
Ten Years of the Hunter Outcome Survey (HOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry Joseph Muenzer1, Simon A
Muenzer et al. Orphanet Journal of Rare Diseases (2017) 12:82 DOI 10.1186/s13023-017-0635-z REVIEW Open Access Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry Joseph Muenzer1, Simon A. Jones2, Anna Tylki-Szymańska3, Paul Harmatz4, Nancy J. Mendelsohn5,6, Nathalie Guffon7, Roberto Giugliani8, Barbara K. Burton9, Maurizio Scarpa10,11, Michael Beck12, Yvonne Jangelind13, Elizabeth Hernberg-Stahl14, Maria Paabøl Larsen15,17, Tom Pulles16,18 and David A. H. Whiteman15* Abstract Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Disease- specific treatment is available in the form of enzyme replacement therapy with intravenous idursulfase (Elaprase®, Shire). Since 2005, the Hunter Outcome Survey (HOS) has collected real-world, long-term data on the safety and effectiveness of this therapy, as well as the natural history of MPS II. Individuals with a confirmed diagnosis of MPS II who are untreated or who are receiving/have received treatment with idursulfase or bone marrow transplant can be enrolled in HOS. A broad range of disease- and treatment-related information is captured in the registry and, over the past decade, data from more than 1000 patients from 124 clinics in 29 countries have been collected. Evidence generated from HOS has helped to improve our understanding of disease progression in both treated and untreated patients and has extended findings from the formal clinical trials of idursulfase. As a long-term, global, observational registry, various challenges relating to data collection, entry, and analysis have been encountered. -
Publications in Scientific Journals (Peer Reviewed) 1
Last Updated July 2020 Publications in Scientific Journals (Peer Reviewed) 1. Eisengart JB, King KE, Shapiro EG, Whitley CB, Muenzer J. The nature and impact of neurobehavioral symptoms in neuronopathic Hunter syndrome. Mol Genet Metab Rep. 2019 Dec 20;22:100549. PMID: 32055445 2. Viskochil D, Clarke LA, Bay L, Keenan H, Muenzer J, Guffon N. Growth patterns for untreated individuals with MPS I: Report from the international MPS I registry. Am J Med Genet A. 2019 Dec;179(12):2425-2432. PMID: 31639289 3. Clarke LA, Giugliani R, Guffon N, Jones SA, Keenan HA, Munoz-Rojas MV, Okuyama T, Viskochil D, Whitley CB, Wijburg FA, Muenzer J. Genotype-phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I Registry. Clin Genet. 2019 Clin Genet. 2019 Oct;96(4):281-289. PMID: 31194252 4. Taylor JL, Clinard K, Powell CM, Rehder C, Young SP, Bali D, Beckloff SE, Gehtland LM, Kemper AR, Lee S, Millington D, Patel HS, Shone SM, Woodell C, Zimmerman SJ, Bailey DB Jr, Muenzer J. The North Carolina Experience with Mucopolysaccharidosis Type I Newborn Screening. J Pediatr. 2019 Aug;211:193-200. PMID: 31133280 5. Akyol MU, Alden TD, Amartino H, Ashworth J, Belani K, Berger KI, Borgo A, Braunlin E, Eto Y, Gold JI, Jester A, Jones SA, Karsli C, Mackenzie W, Marinho DR, McFadyen A, McGill J, Mitchell JJ, Muenzer J, Okuyama T, Orchard PJ, Stevens B, Thomas S, Walker R, Wynn R, Giugliani R, Harmatz P, Hendriksz C, Scarpa M; MPS Consensus Programme Steering Committee; MPS Consensus Programme Co-Chairs. -
Ketoconazole (Systemic) | Memorial Sloan Kettering Cancer Center
PATIENT & CAREGIVER EDUCATION Ketoconazole (Systemic) This information from Lexicomp® explains what you need to know about this medication, including what it’s used for, how to take it, its side effects, and when to call your healthcare provider. Brand Names: Canada APO-Ketoconazole; Ketoconazole-200; TEVA-Ketoconazole Warning This drug is not for use to treat certain types of fungal infections. This includes fungal infections of the skin, nails, or brain. Talk with the doctor. This drug must only be used when other drugs cannot be used or have not worked. Talk with your doctor to be sure that the benefits of this drug are more than the risks. Very bad and sometimes deadly liver problems like the need for a liver transplant have happened with this drug. Some people did not have a raised chance of liver problems before taking this drug. Most of the time, but not always, liver problems have gone back to normal after this drug was stopped. Call your doctor right away if you have signs of liver problems like dark urine, feeling tired, not hungry, upset stomach or stomach pain, light-colored stools, throwing up, or yellow skin or eyes. Blood tests will be needed to watch for any liver problems. Talk with your doctor. Taking this drug with certain other drugs may raise the chance of very bad and sometimes deadly heart problems like a heartbeat that is not normal. Do not take this drug if you are taking any of these drugs: Cisapride, disopyramide, dofetilide, dronedarone, methadone, pimozide, quinidine, or ranolazine. Ketoconazole (Systemic) 1/6 What is this drug used for? It is used to treat fungal infections. -
Treatment of Peripheral Precocious Puberty
View metadata, citation and similar papers at core.ac.uk brought to you by CORE provided by IUPUIScholarWorks Treatment of Peripheral Precocious Puberty Melissa Schoelwer, MD and Erica A Eugster, MD Section of Pediatric Endocrinology, Department of Pediatrics, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana Send correspondence to: 705 Riley Hospital Drive, Room 5960 Indianapolis, IN 46202 Phone: 317-944-3889 Fax: 317-944-3882 Email: [email protected] __________________________________________________________________________________________ This is the author's manuscript of the article published in final edited form as: Schoelwer, M., & Eugster, E. A. (2016). Treatment of Peripheral Precocious Puberty. In Puberty from Bench to Clinic (Vol. 29, pp. 230-239). Karger Publishers. http://dx.doi.org/10.1159/000438895 Peripheral Precocious Puberty Abstract There are many etiologies of peripheral precocious puberty (PPP) with diverse manifestations resulting from exposure to androgens, estrogens, or both. The clinical presentation depends on the underlying process and may be acute or gradual. The primary goals of therapy are to halt pubertal development and restore sex steroids to prepubertal values. Attenuation of linear growth velocity and rate of skeletal maturation in order to maximize height potential are additional considerations for many patients. McCune-Albright syndrome (MAS) and Familial Male-Limited Precocious Puberty (FMPP) represent rare causes of PPP that arise from activating mutations in GNAS1 and the LH receptor gene, respectively. Several different therapeutic approaches have been investigated for both conditions with variable success. Experience to date suggests that the ideal therapy for precocious puberty secondary to MAS in girls remains elusive. In contrast, while the number of treated patients remains small, several successful therapeutic options for FMPP are available. -
CYP3A4 Mediated Pharmacokinetics Drug Interaction Potential of Maha
www.nature.com/scientificreports OPEN CYP3A4 mediated pharmacokinetics drug interaction potential of Maha‑Yogaraj Gugglu and E, Z guggulsterone Sarvesh Sabarathinam1, Satish Kumar Rajappan Chandra2 & Vijayakumar Thangavel Mahalingam1* Maha yogaraja guggulu (MYG) is a classical herbomineral polyherbal formulation being widely used since centuries. The aim of this study was to investigate the efect of MYG formulation and its major constituents E & Z guggulsterone on CYP3A4 mediated metabolism. In vitro inhibition of MYG and Guggulsterone isomers on CYP3A4 was evaluated by high throughput fuorometric assay. Eighteen Adult male Sprague–Dawley rats (200 ± 25 g body weight) were randomly divided into three groups. Group A, Group B and Group C were treated with placebo, MYG and Standard E & Z guggulsterone for 14 days respectively by oral route. On 15th day, midazolam (5 mg/kg) was administered orally to all rats in each group. Blood samples (0.3 mL) were collected from the retro orbital vein at 0.25, 0.5, 0.75, 1, 2, 4, 6, 12 and 24 h of each rat were collected. The fndings from the in vitro & in vivo study proposed that the MYG tablets and its guggulsterone isomers have drug interaction potential when consumed along with conventional drugs which are CYP3A4 substrates. In vivo pharmacokinetic drug interaction study of midazolam pointed out that the MYG tablets and guggulsterone isomers showed an inhibitory activity towards CYP3A4 which may have leads to clinically signifcant interactions. Te use of alternative medicine such as herbal medicines, phytonutrients, ayurvedic products and nutraceuticals used widely by the majority of the patients for their primary healthcare needs. -
Enzyme Replacement Therapy Srx-0019 Policy Type ☒ Medical ☐ Administrative ☐ Payment
MEDICAL POLICY STATEMENT Original Effective Date Next Annual Review Date Last Review / Revision Date 06/15/2011 03/15/2017 10/04/2016 Policy Name Policy Number Enzyme Replacement Therapy SRx-0019 Policy Type ☒ Medical ☐ Administrative ☐ Payment Medical Policy Statements prepared by CSMG Co. and its affiliates (including CareSource) are derived from literature based on and supported by clinical guidelines, nationally recognized utilization and technology assessment guidelines, other medical management industry standards, and published MCO clinical policy guidelines. Medically necessary services include, but are not limited to, those health care services or supplies that are proper and necessary for the diagnosis or treatment of disease, illness, or injury and without which the patient can be expected to suffer prolonged, increased or new morbidity, impairment of function, dysfunction of a body organ or part, or significant pain and discomfort. These services meet the standards of good medical practice in the local area, are the lowest cost alternative, and are not provided mainly for the convenience of the member or provider. Medically necessary services also include those services defined in any Evidence of Coverage documents, Medical Policy Statements, Provider Manuals, Member Handbooks, and/or other policies and procedures. Medical Policy Statements prepared by CSMG Co. and its affiliates (including CareSource) do not ensure an authorization or payment of services. Please refer to the plan contract (often referred to as the Evidence of Coverage) for the service(s) referenced in the Medical Policy Statement. If there is a conflict between the Medical Policy Statement and the plan contract (i.e., Evidence of Coverage), then the plan contract (i.e., Evidence of Coverage) will be the controlling document used to make the determination. -
PROCUR Why Procur Has Been Prescribed for You
Consumer Medicine Information Ask your doctor if you have any questions about PROCUR why Procur has been prescribed for you. Cyproterone acetate 50 mg and 100 mg tablets This medicine is available only with a doctor's prescription. What is in this leaflet Before you take Procur Please read this leaflet carefully before you start taking Procur When you must not take it This leaflet answers some common questions about Procur. It does not contain all the available Do not take Procur if you have an allergy to: information. It does not take the place of talking • any medicine containing cyproterone acetate to your doctor or pharmacist. • any of the ingredients listed at the end of this leaflet All medicines have risks and benefits. Your doctor has weighed the risks of you taking Procur against Some of the symptoms of an allergic reaction may the benefits they expect it will have for you. include: • difficulty in breathing or wheezing If you have any concerns about taking this • shortness of breath medicine, ask your doctor or pharmacist. • swelling of the face, tongue, lips, or other parts of the body Keep this leaflet with the medicine. You may • hives on the skin, rash, or itching need to read it again. Do not take Procur if: What Procur is used for • you are allergic to cyproterone acetate or any other ingredient listed at the end of this leaflet Procur tablets contain the active ingredient • you are pregnant cyproterone acetate. Cyproterone acetate is an • you are breastfeeding antiandrogen. It works by blocking the actions of • you suffer from liver diseases (including sex hormones (androgens) that are produced previous or existing liver tumours, Dubin- mainly in men but also, to a lesser extent in Johnson syndrome or Rotor syndrome) women. -
Studies on the Interactions Between Drugs and Estrogen. III. Inhibitory Effects of 29 Drugs Reported to Induce Gynecomastia on the Glucuronidation of Estradiol
1844 Biol. Pharm. Bull. 27(11) 1844—1849 (2004) Vol. 27, No. 11 Studies on the Interactions between Drugs and Estrogen. III. Inhibitory Effects of 29 Drugs Reported to Induce Gynecomastia on the Glucuronidation of Estradiol a b,1) b b, c Takashi SATOH, Yuki TOMIKAWA, Kaori TAKANASHI, Shinji ITOH, * Shungo ITOH, and b Itsuo YOSHIZAWA a Yakuhan Pharmaceutical Co., Ltd.; Kitahiroshima, Hokkaido 061–1111, Japan: b Hokkaido College of Pharmacy; Otaru, Hokkaido 047–0264, Japan: and c Japan Seamen-Relief-Association Otaru Hospital; 1–7–10 Ironai, Otaru, Hokkaido 047–0031, Japan. Received July 5, 2004; accepted August 27, 2004 To determine the inhibition effects of drugs on the glucuronidation of estradiol (E2), 29 drugs that have been reported to induce gynecomastia were examined in the presence of UDP-glucuronic acid using human hepatic microsomes (pooled) as the enzyme source. The percentage inhibition of the E2 glucuronidation was determined at drug concentrations of 1 mM (approximate therapeutic concentration) and 100 mM (non-clinical overdose con- centration) based on the rate constants for the 3- and 17-glucuronidation of E2 (11.2 and 2.52 pmol/min/mg pro- tein, respectively). The only drug that exhibited 50% or higher inhibition of the 3-glucuronidation at a concen- tration of 1 mM was manidipine (54.4%). When the concentration was 100 mM, manidipine exhibited 100% inhibi- tion of the 3-glucuronidation, and other drugs that exhibited 50% or higher inhibition of the 3-glucuronidation were nicardipine (92%), nisoldipine (90%), nifedipine (84%), domperidone (81%), tacrolimus (80%), nitrendip- ine (77%) and ketoconazole (69%). -
Asfotase Alfa (Strensiq) for Treatment of Hypophosphatasia in Infants and Children
AHRQ Healthcare Horizon Scanning System – Potential High-Impact Interventions Report Priority Area 08: Functional Limitations and Disability Prepared for: Agency for Healthcare Research and Quality U.S. Department of Health and Human Services 5600 Fishers Lane Rockville, MD 20857 www.ahrq.gov Contract No. HHSA290-2010-00006-C Prepared by: ECRI Institute 5200 Butler Pike Plymouth Meeting, PA 19462 December 2015 Statement of Funding and Purpose This report incorporates data collected during implementation of the Agency for Healthcare Research and Quality (AHRQ) Healthcare Horizon Scanning System by ECRI Institute under contract to AHRQ, Rockville, MD (Contract No. HHSA290-2010-00006-C). The findings and conclusions in this document are those of the authors, who are responsible for its content, and do not necessarily represent the views of AHRQ. No statement in this report should be construed as an official position of AHRQ or of the U.S. Department of Health and Human Services. This report’s content should not be construed as either endorsements or rejections of specific interventions. As topics are entered into the System, individual topic profiles are developed for technologies and programs that appear to be close to diffusion into practice in the United States. Those reports are sent to various experts with clinical, health systems, health administration, and/or research backgrounds for comment and opinions about potential for impact. The comments and opinions received are then considered and synthesized by ECRI Institute to identify interventions that experts deemed, through the comment process, to have potential for high impact. Please see the methods section for more details about this process. -
Understanding Drug-Drug Interactions Due to Mechanism-Based Inhibition in Clinical Practice
pharmaceutics Review Mechanisms of CYP450 Inhibition: Understanding Drug-Drug Interactions Due to Mechanism-Based Inhibition in Clinical Practice Malavika Deodhar 1, Sweilem B Al Rihani 1 , Meghan J. Arwood 1, Lucy Darakjian 1, Pamela Dow 1 , Jacques Turgeon 1,2 and Veronique Michaud 1,2,* 1 Tabula Rasa HealthCare Precision Pharmacotherapy Research and Development Institute, Orlando, FL 32827, USA; [email protected] (M.D.); [email protected] (S.B.A.R.); [email protected] (M.J.A.); [email protected] (L.D.); [email protected] (P.D.); [email protected] (J.T.) 2 Faculty of Pharmacy, Université de Montréal, Montreal, QC H3C 3J7, Canada * Correspondence: [email protected]; Tel.: +1-856-938-8697 Received: 5 August 2020; Accepted: 31 August 2020; Published: 4 September 2020 Abstract: In an ageing society, polypharmacy has become a major public health and economic issue. Overuse of medications, especially in patients with chronic diseases, carries major health risks. One common consequence of polypharmacy is the increased emergence of adverse drug events, mainly from drug–drug interactions. The majority of currently available drugs are metabolized by CYP450 enzymes. Interactions due to shared CYP450-mediated metabolic pathways for two or more drugs are frequent, especially through reversible or irreversible CYP450 inhibition. The magnitude of these interactions depends on several factors, including varying affinity and concentration of substrates, time delay between the administration of the drugs, and mechanisms of CYP450 inhibition. Various types of CYP450 inhibition (competitive, non-competitive, mechanism-based) have been observed clinically, and interactions of these types require a distinct clinical management strategy. This review focuses on mechanism-based inhibition, which occurs when a substrate forms a reactive intermediate, creating a stable enzyme–intermediate complex that irreversibly reduces enzyme activity.