…A competitive renewal under this mechanism is possible, but will only be funded once Total funds of an original grant and its renewal will not exceed $200,000 Total funded period of a grant and renewal will not exceed 4 years (excluding NCE) Restrictions apply (see below) Research Tier II Grant Research is more defined than Tier I, and may address but is not limited to the following: Discrete studies or deliverables involving drug repurposing, models, etc. Research needs designed to make possible a significant and new research grant Research needs…
National MPS Society Bylaws Amended April 20, 2024 Bylaws of the National MPS Society The Board of Directors collectively approve the organizational structure, including the nature and flow of responsibilities and authorities, in the Bylaws. They provide for meetings, elections, Officers, financial controls and administrative procedures. They also lay out the form and function of delegation of responsibilities and authorities for the Board of Directors, Officers and Committees. The Bylaws of the National MPS Society describe and define the Organizational Structure of the organization. It documents the delegation of authority…
…the person providing the help is called the guardian and or conservator. A judge makes the determination that the protected person is unable to make independent decisions. You may feel more comfortable obtaining legal advice to establish guardianship and/or conservatorship, however, most states make it easy to do. Laws and terms can vary from state to state: in some states, a guardian is only used to reference personal care, and a conservatorship is limited to finances. It is important to know the laws and terminology of your state. Who can…
…test would be performed to identify higher levels of stored materials. A positive result on both assays, both increased protein markers and elevated specific lysosomal compounds, would need to be found before a family is referred for counseling, additional necessary testing, and therapy. Research regarding Newborn screening for LSDs is increasing. Important questions remain both about the screening process and the testing methods. Controversy will likely continue regarding the appropriateness of screening, especially in the instance of the potential to identify individuals with a condition for which there is no…
…guides research to benefit the patient community. He is a comparative medical geneticist, with over two decades of research experience with the… See More Amber Adams, MS Communications Director Amber is a strategic communicator who has worked with a variety of non-profit and fundraising organizations throughout her career. She has had the unique opportunity to serve a wide array of communities including people who are homeless, people with… See More Vacant Director of Development Click here to learn more about this position and if you might be…
…heart walls (ventricular hypertrophy) is common in MPS IV, most likely because of deposition of storage material within the heart muscle cells. Thickening of mitral and aortic valves is common. Mitral and aortic valves can develop progressive stenosis (narrowing) and/or regurgitation (leaking), although cardiac murmurs may not always be appreciated. Valve replacement has not yet been reported in Morquio Syndrome. Isolated cases of serious interruption of the heart’s conduction system (complete heart block) have been reported and these individuals required pacemakers. Cardiac ultrasound can identify abnormalities of the cardiac valves,…
…a dysmorphology exam. Although it may look different than other exams completed at a doctor’s offices due to how thorough it is, it is not painful and helps answer many questions. The doctor will discuss with you any relevant testing that is available and allow you to decide if you would like to pursue these options. Based upon the medical exam and the information you provided about your child, the geneticist may request x-rays to look for a specific pattern of radiological changes seen in MPS diseases, called dysostosis multiplex….
…Obtaining copies of all pertinent medical records, including knowledge of if the child had HSCT or ERT, prior to the initial evaluation will improve the accuracy of the history and documenting safety precautions that must be observed. The physical therapy examination should include a comprehensive assessment of gross and fine motor abilities using standardized measurement tools in addition to passive joint range of motion, strength testing, joint mobility assessment, endurance testing, and in some cases cardio pulmonary assessment. Children with MPS or ML may have discrepancies in their gross and…
…is progressive osteodystrophy and in whom suitable intravenous access can be established. Baseline tests There are many baseline tests to be done prior to commencing the first infusion of Pamidronate. These tests include blood work, imaging and urine tests, and are important for establishing the need for Bisphosphonate therapy and for tracking the effectiveness in each individual. These tests are outlined in the guidelines for treatment, which are available at www.mpssociety.org • Auxology (hearing tests) Dexa Scan • Full Skeletal Survey • Bone Age (Left wrist and hand) Lateral Spine…
…the patient being transplanted (the “host”) as being foreign. This may be a mild reaction, but in some cases is severe. Transplant protocols have a strategy for the prevention of GvHD, and specific treatments for GvHD are available should GvHD develop. Hair loss, painful mouth sores, and infections, are common side effects or complications associated with the HSCT process. The transplant-related complications can involve virtually any organ or tissue in the body. Of particular concern are serious side effects to the brain, lungs, heart, kidneys and liver. If severe complications…
