MPS clinical trials

Über 7 Millionen englischsprachige Bücher. Jetzt versandkostenfrei bestellen Mehr als 1569 Clinical Trials Stellenangebote verfügbar. Ihre Jobsuche beginnt hier. Suchen Sie unter tausenden von Jobs auf neuvoo, der weltweit grössten Jobbörse The Macular Photocoagulation Study (MPS) consisted of three sets of randomized, controlled clinical trials. Change in best-corrected visual acuity from baseline was the primary outcome for all MPS trials. Other measures of vision are evaluated in each set of trials. The purpose of each is described below This is a longitudinal, prospective, observational, natural history study of patients with MPS IIIA to identify potential surrogate endpoints for future ERT trials via standardized clinical, biochemical, neurocognitive, developmental, behavioral and imaging measures. Other: assessment A maximum of 25 patients with MPS IIIA will be enrolled in the study to provide at least 12 patients who complete the study. This number was not based on any statistical consideration, but is expected to be adequate to describe the natural progression of Sanfilippo A syndrome. Age, Continuous

Human clinical trials involve administering the drug in a controlled environment to gather data on efficacy. This is usually done before making the drug available to all affected patients. The total funds needed to start the FDA approved clinical trial are$2.5 million! The funds collected will cover expenses such as securing the facility where. MPS II is a rare, X-linked, lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase. Because of this deficiency, glycosaminoglycans (GAG) accumulate in multiple tissues and organs, resulting in progressive cellular and organ system dysfunction This study is a multicenter, multiple-dose, dose escalation study designed to evaluate the safety, tolerability, and clinical activity of up to 3 dose levels (10mg,45mg and 90mg monthly for 6 months) of rhHNS administered via an IDDD in patients with Sanfilippo syndrome Type A ages greater than or equal to 3 years of age The National MPS Society recognizes research and clinical trials for MPS and ML exist around the world. We are pleased to share some ongoing clinical trials, with current enrollment, listed below. For more information about ongoing clinical trials, please visit: www.clinicaltrials.gov. Please contact Terri Klein for any additional questions. Hurler Syndrome ( MPS I Continue

MPS III active clinical trials, observational and natural history studies. Clinical trial process. Treatments. There are currently no approved treatments for MPS III. Enzyme replacement therapy (ERT) has not been shown to be effective in MPS III. Bone marrow transplants have been tried on individuals with MPS III but results were not positive Clinical examination and tests to detect excess excretion of mucopolysaccharides in the urine are the first steps in the diagnosis of an MPS disease. Enzyme assays (testing a variety of cells or blood in culture for enzyme deficiency) are needed to provide definitive diagnosis We are committed to funding research to find treatments and cures for those with MPS and ML. The National MPS Society is pleased to announce awards from Cycle I of the 2021 Innovative Research Grants Initiative. A total of $350,000 will be awarded from this cycle. View all MPS News MPS I is a rare genetic condition due to deficiency of the enzyme alpha-l-iduronidase. Laronidase is the manufactured form of the enzyme alpha-l-iduronidase. MPS I is a heterogeneous disease with several clinical phenotypes ranging from the most severe, Hurler syndrome, to the attenuated forms, Hurler-Scheie and Scheie MPS II clinical trials, observational and natural history studies . Clinical trial process. Treatments Enzyme replacement therapy (ERT) Enzyme replacement therapy (ERT) uses an intravenous solution (IV) to replace a deficient or missing enzyme in the body. ERT does not cure the disease but slows its progress by increasing the amount of missing.

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Clinical Trials Registry. ICH GCP. ICH GCP Mucopolysaccharidoses, MPS IV A, MPS VI, MPS - Mucopolysaccharidosis, Morquio A Syndrome, Morquio Syndrome A, Morquio Syndrome . NCT01870375. Completed. Longitudinal Studies of Brain Structure and Function in MPS Disorders MPS I active clinical trials, observational and natural history studies. Clinical trial process. ERT. Enzyme replacement therapy (ERT) uses an intravenous solution (IV) to replace a deficient or missing enzyme in the body. ERT does not cure the disease but slows its progress by increasing the amount of missing enzyme in the body Anakinra is approved by the FDA for treatment of rheumatoid arthritis (RA) and neonatal-onset multisystem inflammatory disease (NOMID). It is not approved for any MPS disorder. The design of this study is an open-label, single center, pilot study of 20 participants with MPS III. There will be an initial screening visit, followed by an 8-week. IRB approval through August 2021. Researchers at the University of Washington are working with the National MPS Society for an important study to further newborn screening efforts. At birth, babies have blood samples collected and tested for genetic conditions. These samples are stored as drops of blood on a card Hunter Syndrome (MPS II) Clinical Trials. A listing of Hunter Syndrome (MPS II) medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area

information to help guide and inform essential aspects of a clinical development program. 3. 102 . 103 Given the rarity of MPS III, a single adequate and well -controlled trial (as described in 21 CF Sanfilippo Syndrome Type A. (MPS IIIA) An open-label, dose-escalation clinical trial assessing the safety of one-time gene therapy ABO-102 for younger, higher-functioning patients with MPS IIIA. An open-label clinical trial assessing the safety of one-time gene therapy ABO-102 for patients with middle and advanced phases of MPS IIIA MPS I, II, and III have early onset clinical phenotypes that affect the brain during development and result in devastating cognitive decline and ultimately death without treatment. Comparisons of outcomes are hindered by diverse protocols and approaches to assessment including applicability to international trials necessary in rare diseases Services include: Providing clinical trial services to pharmaceutical, biotechnology industries and university departments. Setting up and project managing clinical trials performed at Melbourne Pathology. Providing trial and visit specif i c request forms and collection kits. Providing secure data downloads to data management teams

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  1. Inventiva has recently completed Phase IIa of their clinical trial of odiparcil, a drug made for the treatment of mucopolysaccharidosis type VI (MPS VI). Iventiva is very excited about the positive results of their study as MPS VI is a disease with a highly unmet medical need
  2. istration has approved Mepsevii as a treatment for MPS VII, after the drug showed efficacy during clinical trials
  3. The Transpher B Study: A Phase 1/2 Clinical Trial of ABO-101 An open-label, dose-escalation clinical trial assessing the safety of one-time gene therapy ABO-101 for patients with Sanfilippo syndrome type B (MPS IIIB)
  4. Tippi MacKenzie from the University of California San Francisco discussed a clinical trial for in utero ERT in MPS I, II, IVA, VI, and VII, and its potential impact on disease and anti-ERT immune response. There also is a special need for reliable predictors of cognitive disease decline and improvement in neuropathology in MPS syndromes
  5. istering the drug. Phase 2: The study drug is tested to deter

The Transpher A Study: A Phase 1/2 Clinical Trial of ABO-102 An open-label, dose-escalation clinical trial assessing the safety of one-time gene therapy ABO-102 for patients with Sanfilippo syndrome type A (MPS IIIA) This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL310, an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome) Clinical Trials Nct Page Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project

Clinical Trials Registry. ICH GCP. ICH GCP Conditions: Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease . NCT02618512. Terminated Hypothesis: Children diagnosed with a lysosomal disease will exhibit developmental, adaptive, and behavioral strengths and difficulties depending upon 1) biomedical risk factors (i.e. the specific genetic disorder responsible for the illness); 2) available modifying interventions, whether medical or behavioral; and 3) social risks in the children's families, neighborhoods and communities The aim of the study is to assess the efficacy of cardiac MRI to detect coronary disease in diabetic patients. Cardiac MRI will be compared to myocardial scintigraphy with is the method being used in current practice. The investigators believe that cardiac MRI will be as efficient if not better that myocardial scintigraphy to detect tight coronary artery stenosis with the advantage of.

Having had the opportunity to speak at the MPS conference this year, I wanted to put into words what being part of a clinical trial has really meant for us as a family. For those of you who listened to my talk, I was really quite honest, and incredibly optimistic because pretty much at the time, life was kind of plain sailing The main objectives of the 16th International Symposium on MPS and Related Diseases will be: Offering attendees a quality scientific program, with a broad view on new therapeutic possibilities for the future (gene and cell therapies, nanomedicine, etc.), in addition to the review of treatments, updating of clinical trials, and everything. The goal of the MPS program is to provide rigorous training in clinical psychology, including coursework in evidence-based psychological assessment and interventions, as well as state-of-the-art research methods and critical thinking skills. Courses are taught in the evenings at the College Park campus and are designed for working professionals.

Macular Photocoagulation Study (MPS) - Full Text View

We are committed to funding research to find treatments and cures for those with MPS and ML. The National MPS Society is pleased to announce awards from Cycle I of the 2021 Innovative Research Grants Initiative. A total of $350,000 will be awarded from this cycle. View all MPS News It should also be valuable for researchers carrying out clinical trials. MPS IH, MPS II (neuronopathic form), and MPS III are progressive diseases, becoming evident in the second to fourth year of life and ultimately resulting in a loss of attained skills. They are devastating to parents, relentless in their decline, and lethal in the severe forms

A Study of Patients With Sanfilippo Syndrome Type A (MPS

In traditional clinical trial design, efficacy is typically assessed using a single primary endpoint in a randomized controlled trial to detect an expected treatment effect of a therapy in a narrowly selected patient population. The value and application of the MDRI method in two MPS clinical studies. Use of an MDRI post-hoc analysis of the. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 clinical trial in GM1 gangliosidosis is in preparation. In accordance with the. Mucopolysaccharidosis type I (MPS I) is a condition that affects many parts of the body. It is a progressively debilitating disorder; however, the rate of progression varies among affected individuals. You may find these specialists through advocacy organizations, clinical trials, or articles published in medical journals. You may also want. For information about clinical trials being conducted at the NIH Clinical Center in Bethesda, MD, contact the NIH Patient Recruitment Office: Tollfree: (800) 411-1222 TTY: (800) 411-1010 Email: [email protected] Some current clinical trials also are posted on the following page on the NORD website

A prospective, open-label phase 2/3 trial in metastatic pancreatic cancer subjects who have failed two lines of prior systemic therapy. The trial is designed to evaluate the safety and efficacy of SM-88 used with MPS (methoxsalen, phenytoin and sirolimus) in pancreatic cancer and will measure multiple endpoints, including overall survival, progression free survival, relevant biomarkers. MPS IIIA - Lysogene. Mucopolysaccharidosis type IIIA (MPS IIIA), or Sanfilippo syndrome type A, is a rare neurodegenerative lysosomal storage disorder that typically appears in infancy or early childhood. It affects about 1 in 100,000 births and about 2,500 to 3,000 children globally Current Clinical Trials. Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available. Reference There are seven trial locations globally, including one in the United States (California). The others are in Columbia, Germany, Spain, Taiwan, Turkey, and the United Kingdom. Read this clinical trial's information on ClinicalTrials.gov, which includes enrollment criteria, trial locations, and study contact information Through an ongoing proof-of-concept clinical trial, OTL-203 is being evaluated as a potential treatment for patients with the most severe form of MPS-I, known as Hurler syndrome. OTL-203 is an investigational therapy and has not been approved by any regulatory agency or health authority

The Transpher B study, is an open-label, dose-escalation clinical trial evaluating ABO-101 as a treatment for MPS IIIB. Interim results from the study demonstrate that ABO-101 has been well tolerated to date and shown to improve multiple disease biomarkers providing clear evidence of a biologic effect in patients with MPS IIIB Isabella is in the early stages of a clinical trial at CHOC that will evaluate a drug intended to treat a rare pediatric disease called MPS IIIA. The condition is a type of Mucopolysaccharidosis, or MPS, a genetic condition that causes physical abnormalities in young children and causes them to lose their neurological development A major drawback was the high prevalence of neurological involvement and young age of patients in the study cohort compared with the clinical trials. This study emphasizes the limitations of the current tools utilized to monitor ERT efficacy and MPS II disease burden in clinical practice The Lysosomal Disease Network is a team of doctors, nurses, research coordinators, and research labs throughout the U.S., working together to improve the lives of people with this condition through research. The Lysosomal Disease Network has a registry for patients who wish to be contacted about clinical research opportunities

PH MPs feud with Adham over Ivermectin despite ongoing clinical trials. Opposition trio tout drug as Covid-19 miracle cure despite lack of evidence, clash with health minister. Updated 1 day ago · Published on 27 Jul 2021 7:44PM The investigational agent SM-88 demonstrated promising survival in the phase II TYME-88-Panc study in patients with advanced pancreatic cancer. 1,2 The oral modified dysfunctional tyrosine induced a median overall survival (OS) of 6.4 months in patients. The RECIST clinical benefit rate (CBR) of stable disease (SD) or better was 44% with available imaging, and there was a 92% (HR, 0.08;P= .02. It is being used in a clinical study to ensure the drug is safe and effective and the name of the study is Ivermectin treatment efficacy in Covid-19 high-risk patients. It has been approved by the Medical Research and Ethics Committee (MREC) and it is now being carried out in 18 government hospitals Clinical trials assessing dose, safety, and efficacy of intravenous infusion of recombinant human β-glucuronidase in MPS-VII patients are in the recruitment phase or underway (clinicaltrials.gov identifiers NCT01856218, NCT02418455, NCT02230566), following compassionate use of this treatment approach in a single advanced stage-disease patient.

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Iduronate-2-sulfatase Enzyme Replacement Therapy in

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Safety, Tolerability, Ascending Dose and Dose Frequency

Abeona Therapeutics Issues Letter to Stockholders. View Press Release. May 25, 2021. Abeona Therapeutics Appoints Head of Research & Clinical Development. View Press Release. View All About the EngageHunter.com Website EngageHunter.com — the Denali Hunter syndrome (MPS II) community engagement website — is an online destination for emerging information on Denali's scientific advances in Hunter syndrome research and Denali's clinical trials. Visitors who register on the Engage Hunter website will receive updates on. The overarching goal of the NIH Tissue Chip Program is to develop these MPS devices and integrate them to create a Human-on-a-Chip for drug efficacy and safety assessment prior to clinical trials. Current NIH-funded MPS research focuses on modeling various human diseases for efficacy testing (RFA-TR-16-017), and also includes a partnership. Gene therapy clinical trials . Abeona Therapeutics gene therapy for Sanfilippo types A and B. Abeona Therapeutics is conducting clinical trials of gene therapy for Sanfilippo types A and B and currently has sites in the USA, Spain and Australia (type A only). The first trial for Sanfilippo type A started in the USA in May 2016 and the first Australian patients were treated at Adelaide's. Alongside her research and clinical work, Dr. Eisengart has served as an adviser for the Minnesota Department of Health on implementation of newborn screening for MPS I and other rare disorders, and also is a member of Scientific Advisory Board for the National MPS Society USA

Khairy Jamaluddin points out that Singapore's Temasek invested in BioNTech, Israel paid a high price for Pfizer's vaccine, and Indonesia hosted Phase Three clinical trials for Sinovac. Science, Technology and Innovation Minister Khairy Jamaluddin speaks at a press conference after the arrival of the first shipment of Pfizer-BioNTech's Covid. Pfizer/BioNTech and Moderna both started trials of their covid-19 vaccines in children under 12 in March, with results expected in the coming months. On 21 July, Biden told a town hall in Ohio.

The objective of this phase I/II clinical study was to evaluate the efficacy and safety of recombinant human iduronate-2-sulfatase (idursulfase beta, Hunterase®) in the treatment of MPS II. Methods: Thirty-one MPS II patients between 6 and 35 years of age were enrolled in a randomized, single-blinded, active comparator-controlled phase I/II. The third set of MPS clinical trials, the Foveal Study, was designed to determine whether laser photocoagulation was effective for delaying or preventing further visual acuity loss in AMD patients who have subfoveal CNV. Among patients assigned to laser treatment in the Foveal Study, argon laser treatment was compared with krypton red laser. Clinical spectrum of MPS II. MPS II is a variable, progressive, multisystem disorder. In most patients, symptoms are severe and death occurs at an early age. In other patients the disease has a more chronic and protracted course. The age of presentation of MPS II is also variable, as are the presenting signs and disease complications Introduction: Mucopolysaccharidosis type III (MPS III) is a rare disorder characterized by progressive neurocognitive impairment.No disease-modifying treatment is yet available but many are currently under study. The aim of this review is to report on the results of the first clinical trials in MPS III and to discuss specific challenges and recommendations in trial design In addition, we present experimental research with preclinical mRNA and gene editing strategies. Lessons from animal studies and clinical trials have highlighted the importance of an early therapy before extensive neuronal loss. A disease-modifying therapy for MPS III will undoubtedly mandate development of new strategies for early diagnosis

MPS VII is currently in clinical trial. DiFerrante syndrome (mucopolysaccharidosis VIII; MPS VIII) is an obsolete term for a form of MPS described in a single individual with clinical and biochemical features of Morquio and Sanfilippo syndromes. The disorder had been reported to be due to a deficiency of glucosamine-6-sulfate sulfatase This review discusses the best approach to perform MRI in clinical studies, with focus on MPS, and summarizes current knowledge from neuroimaging studies in MPS disorders. Its content is based on information from an expert meeting on the brain in MPS, held on April 28-30, 2016 in Stockholm, Sweden, attended by 39 MPS experts from centers. MPS I Clinical trial and treatment update webinar. Join us for our first MPS I webinar specifically on new treatments. The webinar is for patients, families, carers, and professionals interested in learning more about upcoming treatments and clinical trials. Registration is Closed The study was designed to evaluate safety and efficacy of elosulfase alfa in patients with MPS IVA aged 5 years and older. Areas covered: Outcomes of clinical trials for MPS IVA have been described. Subjects received either 2.0 mg/kg/week, 2.0 mg/kg/every other week, or PBO, for 24 weeks Denali previously announced that Sanofi completed enrollment in a Phase 1b clinical trial of DNL758 (SAR443122), a peripherally-restricted small molecule inhibitor of RIPK1, in hospitalized adult.

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REGENXBIO has the following studies enrolling MPS II patients: Phase I/II clinical trial of RGX-121 in children 4 months to 5 years old This study aims to determine whether RGX-121 is safe, well-tolerated and potentially effective in children over four months and under the age of five years with severe MPS II Mucopolysaccharidosis type IVA (MPS IVA, also called Morquio syndrome, type A) is a metabolic condition that primarily affects the skeleton. The severity, age of onset, and associated symptoms vary significantly from person to person and range from a severe and rapidly progressive, early-onset form to a slowly progressive, later-onset form

MPS III - MPS Societ

  1. The 2021 Clinical Trial Supply forum condenses everything you need to advance your Clinical Trial Supply into two jam packed, interactive days. The industry's most interactive and solution focused CTS forum features deep dive workshops, live polls, panel debates, round table discussions and exclusive key note case studies to ensure that you leave with the industry's best solutions and.
  2. A Phase 2 Clinical Trial to Evaluate the Efficacy and Safety of SM-88 Used With Methoxsalen, Phenytoin, and Sirolimus (MPS) as Response Maintenance Therapy Following Standard Treatments for Patients With Ewing's Sarcoma or as Salvage Therapy for Patients With Clinically Advanced Sarcoma
  3. The Asia-Pacific MPS Registry, an electronic remote data entry system, has been developed by key doctors in the APMN. Rare diseases require international cooperation and collaboration to elucidate their mechanisms and carry out clinical trials; therefore, an organization such as the APMN is required
  4. Clinical trial for Senility , The Effects of Whey vs. Collagen on MPS
  5. We are proud to be the first service of its kind to have grown from a patient organisation - the MPS Society UK. For over a decade, we have led the field in enabling rare disease patients to attend and remain on clinical trials, and access treatments. Advice. We offer specialist expert advice of all aspects of rare diseases
  6. Lysogene's most advanced gene therapy product candidate is LYS-SAF302 (international nonproprietary name [INN]: olenasufligene relduparvovec), currently the subject of AAVance, a global Phase II/III clinical trial for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA), or Sanfilippo syndrome type A

Mucopolysaccharidoses Fact Sheet National Institute of

The first ERT clinical trial in 2001 involved 10 patients with MPS I, treated over 52 weeks with weekly intravenous infusions of 0.58 mg/kg laronidase. The extent of corneal clouding did not change in any of the eight patients with corneal clouding. Several patients reported decreased photophobia or conjunctival irritation The precise etiology of MPS is not clear yet, and further research is needed to determine the root cause. A holistic approach, which blends the basic clinical care with the management of the biopsychosocial model, is essential to patients with MPS to regain their function and improve their quality of life and wellbeing Odiparcil has been evaluated for the treatment of MPS VI adult patients during the iMProveS (improve MPS treatment) Phase IIa clinical trial with positive results published at the end of 2019 The report revealed a mixed picture; the pharmaceutical industry is generally proficient in publishing timely results from its trials, but the MPs reserved criticism for universities and health bodies after finding the results of clinical trials they organise are less likely to be reported The CHOC MPS Multidisciplinary Center team is actively involved in research to further our understanding of MPS and discover the latest treatments and protocols for this disease. Our patients may be invited to participate in clinical trials and studies to try alternate methods of care. Our current research includes: Clinical Trials. A.

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Additional MPS trials in the pipeline. Paradigm is confident a successful phase 2/3 clinical trial will lead to iPPS being registered as a treatment for certain MPS indications, paving the way for commercial success in treating the disease We are encouraged by these interim data and we look forward to continued collaboration with the community to advance MPS II research and DNL310 as a potential treatment for affected individuals.

A Study of Intrathecal Enzyme Therapy for Cognitive

Initial safety and efficacy data from two clinical trials evaluating gene therapies for Sanfilippo syndrome were presented at the ASGCT 23rd Annual Meeting. Sanfilippo syndrome, or mucopolysaccharidosis IIIA (MPS IIIA), is a rare lysosomal storage disease that manifests in young children at a prevalence of about 0.17-2.35 per 100,000 births Clinical research is vital to solving many of today's health challenges, and UPMC Children's Hospital of Pittsburgh is one of the leading sites in the United States for pediatric clinical studies. Children's is one of only a handful of NIH-funded centers in the nation whose clinical trial initiatives include a Pediatric Clinical and Translational Research Center (PCTRC) devoted to pediatric. We are currently evaluating SM-88 in a pivotal trial and an adaptive randomized Phase 2/3 clinical trial (Precision Promise) for patients with second-line pancreatic cancer. Patients are also being enrolled Phase 2 study (HoPES) in high-risk sarcomas In the event of any revision / updation, revised MPS / PMD shall also be shared by FDD /PDD respectively. QA shall ensure the availability of required Technical agreements with 'Clinical Trial Sponsor' and relevant authority

MPS IV - MPS SocietyOdiparcil - Inventiva PharmaCOVID-19 Treatment Updates: Remdesivir’s Coming ToBioMarin Promotes Lon Cardon, PhClinical Trials Staff | Department of Orthopaedic Surgery

Curriculum Overview & Learning Outcomes. The MPS-Clinical Psychological Science is a 30-credit, 10-course graduate program designed to improve your marketable expertise, professional flexibility and mobility, career advancement readiness, and preparation for Ph.D. training. Students receive advanced training in research methods, the ethics. IVA is seeking U.S. and European funding for its NASH and MPS clinical trials. For its lead candidate, the firm has achieved impressive results in its Phase 2b trials, where 'lanifibranor at a. Data that can be aligned with clinical datasets which are accessible (or can be made accessible) through the MPS-Db, e.g., on biomarkers, patient subgroups including pediatric populations, etc. Data that adds to existing datasets or aligns with data already deposited in the MPS-Db, e.g., data generated by the Tissue Chip Testing Centers Day 1: September 16, 2020. Opening remarks: historical overview of myofascial pain syndrome (MPS) Session 1: State of science of MPS: clinical presentation, pathophysiological hypotheses, and challenges. Session 2: Current structural imaging approaches with potential application to MPS. Session 3: Elastography imaging approaches with potential. MPS IX (Hyaluronidase deficiency) is a condition that was first noted in 1996. It is caused by a deficiency of the enzyme, hyaluronidase. This enzyme is used in the lysosome to break down a complex string of sugars known as Glycosaminoglycans (GAGs Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare genetic disorder in which large sugar molecules called glycosaminoglycans (or GAGs or mucopolysaccharides) build up in body tissues. It is a form of lysosomal storage disease.Hunter syndrome is caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S). The lack of this enzyme causes heparan sulfate and.