Orphan neuromuscular diseases

Center for Neuromuscular and Orphan Diseases at the University of FloridaThe multidisciplinary Center for Pediatric Neuromuscular and Orphan Diseases at the University of Florida is founded on the philosophy that integrated, interdisciplinary care is the most effective approach for patients with Neuromuscular and Orphan Diseases.

The Center coordinates and provides patient care, led by leading specialists from diverse medical backgrounds, in one centralized location. Today the center consists of a collaborative effort of specialists with backgrounds in cardiology, general pediatrics, neurology, pulmonary and occupational and physical therapies.

The multidisciplinary Center for Pediatric Neuromuscular and Orphan Diseases provides patient-centric care, allowing patients to interact with all necessary medical providers in one location. This care model is designed to address as many aspects of a patient’s medical needs in a single visit as possible. Established in 2010, and partnering with the Muscular Dystrophy Association, the center operates weekly in the UF Health Orthopaedics and Sports Medicine Institute located in Gainesville, Florida.

Center for Neuromuscular and Orphan Diseases at the University of FloridaAbout Us

The multidisciplinary Center for Pediatric Neuromuscular and Orphan Diseases is a collaborative effort between two colleges and seven divisions or departments at the University of Florida. Five providers have formed a clinical care group to coordinate care for patients with neuromuscular and orphan diseases in need of multidisciplinary care at a single site of service. Clinical services are enhanced by leading research in the field of pediatric neuromuscular diseases, including muscular dystrophy and pompe disease.

Referrals to the center come from throughout the UF Health system and the surrounding communities, and as well as directly from the Muscular Dystrophy Association office located in Jacksonville, Florida.

Center for Neuromuscular and Orphan Diseases at the University of FloridaAny patient who has a neuromuscular disease and would benefit interdisciplinary care is welcomed into the center. We welcome patients from all over the southeastern United States. Following are diseases for which we currently provide patient care:

  • Pompe Disease
  • Distal Muscular Dystrophy
  • Duchenne Muscular Dystrophy
  • Myotonic Dystrophy
  • Becker Muscular Dystrophy
  • Congenital Muscular Dystrophy
  • Facioscapulohumeral Muscular Dystrophy
  • Emery-Dreifuss Dystrophy
  • Freidrich’s Ataxia
  • Barth Syndrome
  • Familial Cardiomyopathy
  • Limb-girdle Dystrophy

Research

Center for Neuromuscular and Orphan Diseases at the University of Florida Center for Neuromuscular and Orphan Diseases at the University of Florida

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"We continue to advance development plans for our gene therapy-based product portfolio for orphan eye diseases including achromatopsia (ACHM) and X-linked retinoschisis (XLRS) and expect to start clinical trials in both programs during the first half ..

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