Congenital Fiber-Type Disproportion
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Congenital Fiber-Type Disproportion Overview

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Learn About Congenital Fiber-Type Disproportion

Condition 101 content is not available at this time, but we are continually updating the site. Please check back.

However, there may be experts who have treated this or similar conditions in our Find a Doctor section and research may be available in our Latest Advances section.

Who are the top Congenital Fiber-Type Disproportion Local Doctors?
Elite in Congenital Fiber-Type Disproportion
Elite in Congenital Fiber-Type Disproportion

Penn Neuroscience Center - Neurology

3400 Civic Center Boulevard, South Pavilion, 2nd Floor, 
Philadelphia, PA 
Languages Spoken:
English
Accepting New Patients

James Dowling is a Neurologist practicing medicine in Philadelphia, Pennsylvania. Dr. Dowling is rated as an Elite provider by MediFind in the treatment of Congenital Fiber-Type Disproportion. He is also highly rated in 20 other conditions, according to our data. His clinical expertise encompasses Tubular Aggregate Myopathy, Congenital Fiber-Type Disproportion, X-Linked Myotubular Myopathy, and Horizontal Gaze Palsy with Progressive Scoliosis. Dr. Dowling is board certified in Neurology - Child Neurology, 2007. Dr. Dowling is currently accepting new patients.

Elite in Congenital Fiber-Type Disproportion
Pediatric Neurology
Elite in Congenital Fiber-Type Disproportion
Pediatric Neurology

Office

3401 Civic Center Blvd, 
Philadelphia, PA 
Languages Spoken:
English

Carsten Bonnemann is a Pediatric Neurologist practicing medicine in Philadelphia, Pennsylvania. Dr. Bonnemann is rated as an Elite provider by MediFind in the treatment of Congenital Fiber-Type Disproportion. He is also highly rated in 21 other conditions, according to our data. His clinical expertise encompasses Tubular Aggregate Myopathy, Congenital Fiber-Type Disproportion, X-Linked Myotubular Myopathy, Centronuclear Myopathy, and Gastrostomy.

 
 
 
 
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Elite in Congenital Fiber-Type Disproportion
Elite in Congenital Fiber-Type Disproportion
Illkirch-graffenstaden, FR 

Johann Bohm practices practicing medicine in Illkirch-graffenstaden, France. Bohm is rated as an Elite expert by MediFind in the treatment of Congenital Fiber-Type Disproportion. They are also highly rated in 13 other conditions, according to our data. Their clinical expertise encompasses Tubular Aggregate Myopathy, X-Linked Myotubular Myopathy, Congenital Fiber-Type Disproportion, and Stormorken Syndrome.

What are the latest Congenital Fiber-Type Disproportion Clinical Trials?
A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants With Invasive Ventilator-dependent X-linked Myotubular Myopathy

Summary: X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM. The protein myotubularin is needed for muscle development, movement and breathing. A gene called MTM1 tells the body to make myotubularin. XLMTM is caused by changes, or mutations, in the MTM1 gene. Changes in the MTM1 ge...

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A Non-interventional, Epidemiologic Study of XLMTM and Clinical Expression in the Liver

Summary: XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness. There is currently no cure for XLMTM. Ongoing care is needed to manage symptoms and prevent further medical problems from this condition. Recent research shows that individuals with XLMTM often have...