Question: Stem Cell Therapy for Muccular Dystrophy I have read the Medline Abstracts 98,99 of 'Limb-girdle muscular dystrophy'. Please could you tell me how I contact a specialist to enquire further with this...Thank you...

Reference:
Limb-girdle muscular dystrophy: Limb-girdle muscular dystrophy is a group of disorders which affect the voluntary muscles around the hips and shoulders. The conditions are progressive, leading to a loss of muscle strength and bulk over a number of years. Onset may occur in childhood, adolescence, young adulthood, or even later. Males and females are affected in equal numbers. [1] Most forms of limb girdle muscular dystrophy are inherited in an autosomal recessive manner. Several rare forms are inherited in an  autosomal dominant pattern. [2] While there are no treatments which directly reverse the muscle weakness associated with this condition, supportive treatment can decrease the complications. [3] There are at least 20 different types of limb-girdle muscular dystrophy. [1] Limb-girdle muscular dystrophy (LGMD) is most often inherited in an  autosomal recessive manner; less commonly, rare sub-types may be inherited in an  autosomal dominant manner.  There may be difficulties diagnosing the condition accurately, and often the mode of inheritance cannot be determined. Therefore, it may be challenging to determine the exact recurrence risks for some families. Establishing the type of LGMD in an affected individual can be useful for discussing the clinical course of the disease as well as for determining who else in the family may be at risk for the condition. [4] Making a diagnosis for a genetic or rare disease can often be challenging. Healthcare professionals typically look at a person’s medical history, symptoms, physical exam, and laboratory test results in order to make a diagnosis. The following resources provide information relating to diagnosis and testing for this condition. If you have questions about getting a diagnosis, you should contact a healthcare professional. Testing Resources Orphanet lists international laboratories offering diagnostic testing for this condition. Unfortunately, no definitive treatments for LGMD exist. Management depends on each individual and the specific type of LGMD that the individual has. However, the American Academy of Neurology has developed guidelines for treatment of LGMD including: [5] [6] Weight control to avoid obesity Physical therapy and stretching exercises to promote mobility and prevent contractures (fixed tightening of the muscles) Use of mechanical aids such as canes, walkers, orthotics, and wheelchairs as needed to help ambulation and mobility Monitoring and surgical intervention as needed for orthopedic complications, such as foot deformity and scoliosis cardiomyopathy A team approach to treatment is recommended including a neurologist , pulmonologist , cardiologist , orthopedic surgeon, physiatrist , physical/occupational/ speech therapist , nutritionist , orthopedist , mental health counselors, and geneticist / genetic counselor . [5] [6] While not a currently available treatment option, some studies have shown  promising results with the use of gene therapy . More research is needed to prove the safety and efficacy of this treatment approach. [6] Management Guidelines The American Academy of Neurology (AAN), the medical specialty society of neurologists, offers a summary of recommended guidelines for Limb-girdle muscular dystrophy  GeneReviews provides current, expert-authored, peer-reviewed, full-text articles describing the application of genetic testing to the diagnosis, management, and genetic counseling of patients with specific inherited conditions. Project OrphanAnesthesia is a project whose aim is to create peer-reviewed, readily accessible guidelines for patients with rare diseases and for the anesthesiologists caring for them. The project is a collaborative effort of the German Society of Anesthesiology and Intensive Care, Orphanet, the European Society of Pediatric Anesthesia, anesthetists and rare disease experts with the aim to contribute to patient safety. The following diseases are related to Limb-girdle muscular dystrophy. If you have a question about any of these diseases, you can contact GARD. Epidermolysa bullosa simplex with muscular dystrophy Limb-girdle muscular dystrophy type 1A Limb-girdle muscular dystrophy type 1B Limb-girdle muscular dystrophy type 1C Limb-girdle muscular dystrophy type 1D Limb-girdle muscular dystrophy type 1E Limb-girdle muscular dystrophy type 1F Limb-girdle muscular dystrophy type 1G Limb-girdle muscular dystrophy type 1H Limb-girdle muscular dystrophy type 2A Limb-girdle muscular dystrophy type 2E Limb-girdle muscular dystrophy type 2F Limb-girdle muscular dystrophy type 2H Limb-girdle muscular dystrophy type 2I Limb-girdle muscular dystrophy type 2J Limb-girdle muscular dystrophy type 2K Limb-girdle muscular dystrophy type 2L Limb-girdle muscular dystrophy type 2M Limb-girdle muscular dystrophy type 2N Limb-girdle muscular dystrophy type 2O Limb-girdle muscular dystrophy type 2Q Limb-girdle muscular dystrophy type 2S Limb-girdle muscular dystrophy type 2T Limb-girdle muscular dystrophy, type 2B Limb-girdle muscular dystrophy, type 2C Limb-girdle muscular dystrophy, type 2D Limb-girdle muscular dystrophy, type 2G Muscular dystrophy Progressive muscular dystrophy

Answer: Management  of Limb-girdle muscular dystrophy  depends on each individual and the specific type of LGMD. The  guidelines for treatment of LGMD developed by the American Academy of Neurology   include: weight control to avoid obesity, physical therapy and stretching exercises to promote mobility and prevent tightening of the muscles, use of mechanical aids such as canes, walkers, orthotics, and wheelchairs as needed to help ambulation and mobility Monitoring and surgical intervention as needed for orthopedic complications, such as foot deformity and scoliosis cardiomyopathy A team approach to treatment is recommended including a neurologist , pulmonologist , cardiologist , orthopedic surgeon, physiatrist , physical/occupational/ speech therapist , nutritionist , orthopedist , mental health counselors, and geneticist / genetic counselor . While not a currently available treatment option, some studies have shown  promising results with the use of gene therapy . More research is needed to prove the safety and efficacy of this treatment approach.