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60-572: MDAA is an acronym that may refer to: Muscular Dystrophy Association , formerly known as the Muscular Dystrophy Associations of America (MDAA) Missile Defense Advocacy Alliance , an organization advocating the development and deployment of missile defense for the United States and its allies Mutual Defense Assistance Act , a 1949 United States Act of Congress Topics referred to by

120-403: A big charity to take care of or cure them." Clinical data management Clinical data management ( CDM ) is a critical process in clinical research , which leads to generation of high-quality, reliable, and statistically sound data from clinical trials . Clinical data management ensures collection, integration and availability of data at appropriate quality and cost. It also supports

180-669: A bill that would amend the Public Health Service Act to revise the muscular dystrophy research program of the National Institutes of Health (NIH). MDA argued that "a great deal of work still needs to be done, and increased federal support is needed to ensure that researchers can continue making progress toward finding a cure." In December 2021, President Joe Biden signed the Accelerating Access to Critical Therapies for ALS Act into law. The act aimed to speed up

240-713: A clinical trial may be sent to a single central laboratory for analysis. The clinical data manager liaises with the central laboratory and agrees data formats and transfer schedules in Data Transfer Agreement. The sample collection date and time may be reconciled against the CRF to ensure that all samples collected have been analysed. Analysis of clinical trial data may be carried out by laboratories, image processing specialists or other third parties. The clinical data manager liaises with such data providers and agree data formats and transfer schedules. Data may be reconciled against

300-480: A clinical trial utilizing a paper CRF, the relational database is built separately. In both cases, the relational database allows entry of all data captured on the Case report form. All computer systems used in the processing and management of clinical trial data must undergo validation testing to ensure that they perform as intended and that results are reproducible. The Clinical Data Interchange Standards Consortium leads

360-505: A different approach to CRF data as, for example, it is generally not practical to raise data queries. Patient diaries may be developed in either paper or electronic (eDiary) formats. Such eDiaries generally take the form of a handheld device which enables the subject to enter the required data and transmits this data to a centralised server. Once all expected data is accounted for, all data queries closed, all external data received and reconciled and all other data management activities complete

420-478: A funding commitment of more than $ 66 million. MDA is actively involved in supporting the development of gene therapies for rare neuromuscular diseases. In 2023, the association introduced the Kickstart Program to lower the barriers for gene therapy development. The program assembles a team of experts to collaboratively assist selected projects, positioning them for follow-on grant funding. The Kickstart Program

480-407: A key role in the setup and conduct of a clinical trial. The data collected during a clinical trial form the basis of subsequent safety and efficacy analysis which in turn drive decision making on product development in the pharmaceutical industry. The clinical data manager is involved in early discussions about data collection options and then oversees development of data collection tools based on

540-442: A paper CRF the pages are entered by data entry operators. Best practice is for a first pass data entry to be completed followed by a second pass or verification step by an independent operator. Any discrepancies between the first and second pass may be resolved such that the data entered is a true reflection of that recorded on the CRF. Where the operator is unable to read the entry the clinical data manager should be notified so that

600-688: A quarterly magazine, Quest, catering to people with neuromuscular diseases and their caretakers. Mindy Henderson, diagnosed with type 2 spinal muscular atrophy (SMA) and a patient advocate, is the editor-in-chief as of 2023. The magazine, with a circulation of around 100,000, extends its reach through distribution at neurologists' offices and the MDA's network of 150 care centers across the country. Besides focusing on science and research, Quest's coverage includes adaptive lifestyles, travel, medical equipment, accessible fashion, employment, and media representation. Every summer, for one week, thousands of children from across

660-669: A second national sponsor of the organization. In 1982, the MDA Shamrocks program launched in Grand Rapids, Michigan , and became a national program one year later. In 1996, MDA and Lewis were jointly honored by the American Medical Association with a lifetime achievement awards for their contributions to the health and welfare of humanity. Dr. Stanley Appel established Houston Methodist Hospital's amyotrophic lateral sclerosis clinic with MDA in 1982, which has evolved into

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720-513: A severe form of muscular dystrophy, ALS (amyotrophic lateral sclerosis), and Charcot–Marie–Tooth disease (CMT), which impacts peripheral nerves. MDA also addresses numerous rare disorders, such as Andersen–Tawil syndrome , Laing distal myopathy , and Walker–Warburg syndrome . Additionally, the association covers conditions like Becker muscular dystrophy (BMD), congenital muscular dystrophies (CMD), myotonic dystrophy (DM), spinal muscular atrophy (SMA), and myasthenia gravis (MG). MDA supported

780-658: A six-year hiatus. The two-hour event was held virtually due to the COVID-19 pandemic , and streamed live on the Laugh Out Loud network and its YouTube channel. Celebrity guests for the 2020 telethon included Jack Black , Josh Gad , Michael B. Jordan , and Jillian Mercado . In November 2020, MDA launched a tool, called the neuroMuscular ObserVational Research (MOVR) Visualization and Reporting Platform (VRP), to help make clinical data more accessible and accelerate discovery of muscular dystrophy treatments. In response to

840-830: A tradition of selecting National Ambassadors who are people living with neuromuscular disease. Since the program's inception in 1952, over 45 National Ambassadors have shared their experiences to emphasize the need for funding research and developing treatments and cures for neuromuscular diseases. These representatives engage with partners, volunteers, supporters, and notable figures, gaining exposure through social media channels and national media outlets. Former youth ambassadors have pursued diverse fields in adulthood, advocating for improvements in accessibility, early intervention treatments through newborn screening, and advancements in research and care for MDA families and individuals living with disabilities. MDA's National Ambassadors were formerly known as "National Goodwill Ambassadors", which, until

900-408: A validation check detects a problem with the data. Validation checks are run automatically whenever a page is saved “submitted” and can identify problems with a single variable, between two or more variables on the same eCRF page, or between variables on different pages. A variable can have multiple validation checks associated with it. Errors can be resolved in several ways: Samples collected during

960-465: Is Ethan LyBrand, who has Duchenne muscular dystrophy and gained national recognition for his Shorty Award-winning "Joke-A-Day for MDA" campaign during the COVID-19 pandemic. In 1986, MDA-funded researcher Louis M. Kunkel identified the dystrophin gene, the gene for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). MDA funded the first gene therapy trial in 1999, followed by

1020-467: Is built, the clinical data manager (and other parties as appropriate) conducts User Acceptance Testing (UAT). The tester enters test data into the e-CRF and record whether it functions as intended. UAT is performed until all the issues (if found) are resolved. When an electronic CRF is in use, data entry is carried out at the investigative site where the clinical trial is conducted by site staff who have been granted appropriate access to do so. When using

1080-450: Is different from Wikidata All article disambiguation pages All disambiguation pages Muscular Dystrophy Association The organization was founded in 1950 by a group with personal connections to muscular dystrophy , including Paul Cohen who lived with the disease. Originally known as the Muscular Dystrophy Associations of America, it was renamed to its present name in the 1970s. In 1954, MDA began its partnership with

1140-527: Is entered, to map certain data points from one CRF to the other, to calculate certain fields like Subject's Age, BMI etc.. Edit checks help the investigators to enter the right data right at the moment data is entered and also help in increasing the quality of the Clinical trial data. For a clinical trial utilizing an electronic CRF, database design and CRF design are closely linked. The electronic CRF enables entry of data into an underlying relational database. For

1200-576: Is recognized as the largest network of multidisciplinary clinics specializing in neuromuscular diseases in the United States, with over 150 clinics distributed throughout the country. In addition to offering specialized care, these centers are also active sites of research. In 2023, MDA expanded its Resource Center by launching the Gene Therapy Support Network. This network provides resources and guidance about approved gene therapies for people living with neuromuscular diseases, aiming to help

1260-609: Is recognized by the Better Business Bureau's Wise Giving Alliance as an accredited charity that meets all 20 Standards for Charity Accountability. As of the year ending December 31, 2022, MDA reported significant funding directed towards patient and community services, professional public health education, and research. The organization's financial activities, leadership compensation, and fundraising methods are transparently disclosed, emphasizing their commitment to accountability and ethical practices. Charity Navigator , which

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1320-415: Is retained at the investigative site. When an item or variable has an error or a query raised against it, it is said to have a “discrepancy” or “query”. All EDC systems have a discrepancy management tool or also refer to “edit check” or “validation check” that is programmed using any known programming language (e.g. SAS, PL/SQL, C#, SQL, Python, etc). So what is a ‘query’? A query is an error generated when

1380-463: Is the data collection tool for the clinical trial and can be paper or electronic. Paper CRFs will be printed, often using No Carbon Required paper, and shipped to the investigative sites conducting the clinical trial for completion after which they are couriered back to Data Management. Electronic CRFs enable data to be typed directly into fields using a computer and transmitted electronically to Data Management. Design of CRFs needs to take into account

1440-653: Is the largest independent evaluator of charities, gives MDA two out of four stars based on Financial, Accountability, and Transparency Performance Metrics. In 2019, CharityWatch gave MDA a grade of D, citing many financial issues in the organization. MDA has received the Candid Platinum Seal of Transparency from GuideStar , recognizing its commitment to transparency and accountability. MDA and Lewis have been criticized by disability rights activists for their tendency to paint disabled people as, these advocates say, "pitiable victims who want and need nothing more than

1500-575: The International Association of Fire Fighters for its annual Fill the Boot fundraising drive. In 1955, the organization held its first summer camp. In 1980, American motorcycle manufacturer Harley Davidson became an MDA National Sponsor; in 1987 the MDA Ride For Life program began, a fundraising motorcycle ride held over Labor Day weekend. In 1986, oil and gas provider Citgo became

1560-617: The Newborn Screening Saves Lives Reauthorization Act of 2013 (H.R. 1281; 113th Congress) , a bill that would amend the Public Health Service Act to reauthorize grant programs and other initiatives to promote expanded screening of newborns and children for heritable disorders. The MDA argued that "many of the drug therapies currently under development for MDA's community will be of most benefit if administered either presymptomatically or early in

1620-494: The 1980s, were referred to as "poster children". In 1952, the MDA inaugurated Michael Danna as its first Poster Child. One of the most well-known ambassadors was Mattie Stepanek , the National Goodwill Ambassador from 2002 until his death in 2004, notable for his best-selling Heartsongs series of poetry books, and his appearances on The Oprah Winfrey Show and Good Morning America. Another notable ambassador

1680-428: The COVID-19 pandemic, MDA converted several of its traditional programs to virtual formats and introduced new virtual programming. The latter included several Facebook Live events discussing challenges that the pandemic placed on people with disabilities. It also provided COVID-19 resources and recommendations for neuromuscular patients and providers via its online resource center. Throughout stay at home orders due to

1740-570: The COVID-19 pandemic, MDA shared a "joke of the day" from National Ambassador Ethan Lybrand via their social media channels. In January 2021, MDA announced NFL running back Nyheim Hines as its national spokesperson. The Muscular Dystrophy Association (MDA) was established to lead research and support for individuals affected by muscular dystrophy and related diseases. In a recent [interview]( https://denver-frederick.com/2024/01/12/the-muscular-dystrophy-association-reimagined-a-chat-with-mdas-dr-donald-wood/ ) with Denver Frederick, Dr. Donald Wood,

1800-453: The CRF to ensure consistency. The CRF collects adverse events reported during the conduct of the clinical trial however there is a separate process which ensures that serious adverse events are reported quickly. The clinical data manager must ensure that data is reconciled between these processes. Where the subject is required to record data (e.g. daily symptoms) then a diary is provided for completion. Data management of this data requires

1860-707: The MDA ALS Research and Clinical Center at the Houston Methodist Neurological Institute. Dr. Appel's pioneering work includes the development of an immunotherapy treatment that may slow the progression of ALS. Dr. Appel's efforts were recognized by the MDA when he received the MDA's Tribute Award in 2022. In October 2020, the MDA Telethon , which had originally run from 1966 to 2014, was reimagined as The MDA Kevin Hart Kids Telethon after

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1920-451: The MDA announced that Lewis resigned as host and chairman, due to circumstances not revealed. However, in 2016, one year before his death, Lewis broke a five-year silence in a video endorsing MDA's redesigned web site and brand, declaring that the work to end muscular dystrophy be continued. Additionally, Lewis' support was so ironclad over the years that children and adults assisted by MDA are referred to as Jerry's Kids . From 2012 to 2014,

1980-513: The United States to raise money for research and patient services. In 2022, MDA resumed in-person Muscle Walks in numerous locations, including Phoenix, Chicago, St. Louis, Detroit, Boston, Twin Cities, Houston, and Dallas, while continuing to offer a virtual Muscle Walk option to enhance participation flexibility. Throughout February and March, retailers across the US contribute to the MDA Shamrocks campaign,

2040-537: The United States. The partnership between MDA and IAFF dates back to 1954, with IAFF designating MDA as its charity of choice. Firefighters across the country have raised $ 690 million over nearly seven decades, leading to breakthroughs in research, FDA-approved treatments, and advocacy efforts such as access to care and newborn screening. MDA's annual payments to IAFF, which ranged from $ 980,000 to $ 1.4 million between 2015 and 2020 according to U.S. Department of Labor filings, received some scrutiny for their size relative to

2100-446: The charity's expenditures. Debuting in 1966 and held annually on Labor Day weekend until 2014, the telethon was originally hosted by veteran film star, comedian and singer Jerry Lewis , who also served as the MDA's national chairman since its inception in 1950 and hosted the show until 2010. In 2005, the MDA made the unprecedented decision to pledge $ 1 million of the telethon's money raised to Hurricane Katrina disaster relief , making

2160-414: The clinical trial protocol. Once subject enrollment begins, the data manager ensures that data are collected, validated, complete, and consistent. The clinical data manager liaises with other data providers (e.g. a central laboratory processing blood samples collected) and ensures that such data are transmitted securely and are consistent with other data collected in the clinical trial. At the completion of

2220-438: The clinical trial, the clinical data manager ensures that all data expected to be captured have been accounted for and that all data management activities are complete. At this stage, the data are declared final (terminology varies, but common descriptions are "Database Lock", “Data Lock” and "Database Freeze"), and the clinical data manager transfers data for statistical analysis . Standard operating procedures (SOPs) describe

2280-593: The community navigate the complexities of emerging gene therapies. Clinical networking through the MDA Care Center Network contributes additional support by facilitating best practice sharing between different care centers. MDA targets a wide range of neuromuscular diseases, including muscular dystrophies, motor neuron diseases , ion channel diseases, mitochondrial diseases , myopathies , neuromuscular junction diseases , and peripheral nerve diseases. Specific examples include Duchenne muscular dystrophy (DMD),

2340-543: The conduct, management and analysis of studies across the spectrum of clinical research as defined by the National Institutes of Health (NIH). The ultimate goal of CDM is to ensure that conclusions drawn from research are well supported by the data. Achieving this goal protects public health and increases confidence in marketed therapeutics. Job profile acceptable in CDM: clinical researcher, clinical research associate, clinical research coordinator etc. The clinical data manager plays

2400-553: The country who have been diagnosed with neuromuscular diseases are able to attend a camp designated for only them. MDA Summer Camp offers children and young adults the opportunity to participate in recreational activities that foster independence, self-confidence, skill development, and friendships. There is a one counselor to one camper ratio and the entire week the children, ages 8–17, are paired with an adult volunteer. They participate in activities and games and stay overnight. The camps are set up locally and are different weeks throughout

2460-531: The current CEO of MDA, discussed the organization's founding legacy and its evolving role in reimagining support and research for the muscular dystrophy community. Dr. Wood highlighted MDA's commitment to continuing the vision of its founders through expanded programs and initiatives. Research!America Advocacy Award (2025) In 2025, MDA received the Research!America Advocacy Award in recognition of its work in research and advocacy for neuromuscular diseases. MDA has

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2520-679: The development and approval of therapies for ALS and other neuromuscular diseases. During the signing ceremony President Biden acknowledged the Muscular Dystrophy Association for playing a crucial role in advocating for the legislation. In May 2024, MDA supported the reauthorization of the Federal Aviation Administration (FAA) through a $ 105 billion bill passed by Congress. The legislation includes improvements in air travel accessibility for people with disabilities, representing significant progress in this area. MDA

2580-434: The development of global, system independent data standards which are now commonly used as the underlying data structures for clinical trial data. These describe parameters such as the name, length and format of each data field (variable) in the relational database. Validation Rules are electronic checks defined in advance which ensure the completeness and consistency of the clinical trial data. Once an electronic CRF (eCRF)

2640-630: The development of improved care and treatment for the neuromuscular community. The conference also hosts the Annual Insights in Research Investment Summit, which focuses on research and investment stakeholders in neuromuscular diseases. The 2023 conference featured a keynote address by Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, on the growing impact of gene therapies in treating rare disorders. MDA publishes

2700-594: The donation specifically to the Salvation Army (though the telethon also urged viewers to give to the American Red Cross ). In 2008, the annual televised fundraiser raised a record $ 65,031,393. Originally broadcast for up to 21½ hours from 1966 to 2010, the event was cut back to six hours in 2011. The 2011 edition of the telethon was originally announced to have been Lewis' last as host, with him continuing his role as national chairman; however, on August 3, 2011,

2760-400: The entry may be clarified with the person who completed the CRF. Data validation is the application of validation rules to the data. For electronic CRFs the validation rules may be applied in real time at the point of entry. Offline validation may still be required (e.g. for cross checks between data types) Where data entered does not pass validation rules then a data query may be issued to

2820-456: The event was broadcast October 24, 2020, at 8 p.m. EDT. The special was a dual-charity event, with proceeds going towards both MDA and "Help From The Hart", an organization founded by the aforementioned new host of the program. Hart's charity announced that it will use its portion of funds raised to "support education, health and social needs programs targeting underserved communities and servicing youth through education scholarships." The telethon

2880-646: The first vector based gene therapy trial for DMD in 2006. In 2007, MDA funded Adrian R. Krainer and his colleagues at the Cold Spring Harbor Laboratory in Laurel Hollow , New York for the early-stage development of nusinersen . Nusinersen became FDA approved in 2016 as the first treatment for spinal muscular dystrophy (SMA). As of 2018, MDA had a total funding commitment of more than $ 58 million distributed among 312 research grants. By 2019, MDA supported 252 research projects worldwide, totaling

2940-425: The information required to be collected by the clinical trial protocol and intended to be included in statistical analysis. Where available, standard CRF pages may be re-used for collection of data which is common across most clinical trials e.g. subject demographics. Apart from CRF design, electronic trial design also includes edit check programming. Edit checks are used to fire a query message when discrepant data

3000-408: The investigative site where the clinical trial is conducted to request clarification of the entry. Data queries must not be leading (i.e. they must not suggest the correction that should be made). For electronic CRFs only the site staff with appropriate access may modify data entries. For paper CRFs, the clinical data manager applies the data query response to the database and a copy of the data query

3060-438: The largest St. Patrick's Day fundraising initiative in the country. They provide customers with the opportunity to either round up their purchases or donate a specific amount, such as $ 1, $ 3, or $ 5. Customers can write their names on paper shamrocks, which are then displayed in stores to show support for MDA's mission to empower people with neuromuscular diseases to lead longer, more independent lives. MDA's Care Center Network

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3120-490: The months of May through August. The entire camp staff are volunteer members and are required to interview and apply with good recommendations. The camp is offered at no cost to campers and their families; the cost of the camp for the campers and volunteers is covered by the many fundraisers the MDA does each year. Started in 2010, the MDA Muscle Walk is an annual 1 to 3.1 mile lap event held in over 150 communities across

3180-626: The process to be followed in conducting data management activities and support the obligation to follow applicable laws and guidelines (e.g. ICH GCP and 21CFR Part 11 ) in the conduct of data management activities. The data management plan describes the activities to be conducted in the course of processing data. Key topics to cover include the SOPs to be followed, the clinical data management system (CDMS) to be used, description of data sources, data handling processes, data transfer formats and process, and quality control procedure The case report form (CRF)

3240-575: The production of a protein called micro-dystrophin in patients' muscles. Advancements in Duchenne muscular dystrophy treatment have prompted MDA to lobby for newborn screening panels for early detection and treatment initiation. During National Boot Day, the MDA partners with the International Association of Fire Fighters (IAFF) to launch Fill the Boot fundraising events. These events help support research, care, and advocacy for families affected by muscular dystrophy, ALS, and related neuromuscular diseases in

3300-462: The progression of the disease. Thus, for some of the diseases in MDA's program, the availability of a newborn screening program at the time of treatment availability presents the best opportunity for impacting optimal and potential lifesaving treatment outcomes." MDA supported the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2013 (H.R. 594; 113th Congress) ,

3360-405: The same term [REDACTED] This disambiguation page lists articles associated with the title MDAA . If an internal link led you here, you may wish to change the link to point directly to the intended article. Retrieved from " https://en.wikipedia.org/w/index.php?title=MDAA&oldid=1155148439 " Category : Disambiguation pages Hidden categories: Short description

3420-495: The show was known as the MDA Show of Strength . In early 2015, the organization announced it was discontinuing the show. On September 9, 2020, MDA executives announced plans to relaunch their annual MDA telethon with a new host, comedian Kevin Hart . Entitled The MDA Kevin Hart Kids Telethon , the new two-hour telethon was seen exclusively through participating social media platforms;

3480-483: Was MDA's 70th Anniversary Show, hosted by entertainment TV journalists Nancy O'Dell and Jann Carl . The MDA Clinical & Scientific Conference is a prominent event that gathers over 2,000 professionals, including scientists and clinicians, specializing in neuromuscular diseases. The conference showcases research, fosters collaborations, and promotes the latest advancements in the field. It covers various aspects of research, from pre-clinical to clinical, and supports

3540-493: Was accompanied by a 10-hour Let's Play livestream , called Let's Play For A Cure, which featured DJ and producer Zedd , gaming personality missharvey and esports athletes. The livestream was a part of a week-long "streamathon", which featured esports stars playing games including League of Legends , Ro cket Lea gue , Fortnite , Call of Duty and Fall Guys . Following the Kevin Hart Kids Telethon

3600-418: Was presented at the 2023 MDA Clinical & Scientific Conference, where discussions focused on opportunities to de-risk gene therapies, current barriers to development, collaborative frameworks, and data management. MDA supported the FDA's decision in 2023 to approve an experimental gene therapy developed by Sarepta Therapeutics that aims to slow the progression of Duchenne muscular dystrophy by facilitating

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