Duchenne Muscular Dystrophy Clinical Trial
Official title:
Study Safety and Efficacy of of AUTOLOGOUS Bone Marrow Derived Mono Nuclear Stem Cell (BMMNCs) for the Patient With Duchenne Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial
This Study is single arm, single center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy,
Muscular dystrophies, or MD, are a group of inherited conditions, which means they are
passed down through families. They may occur in childhood or adulthood. There are many
different types of muscular dystrophy. They include:
Duchenne muscular dystrophy is a form of muscular dystrophy that worsens quickly. Other
muscular dystrophy (including Becker's muscular dystrophy) get worse much more slowly.
Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the
muscles). However, it often occurs in people without a known family history of the
condition.
Symptoms usually appear before age 6 and may appear as early as infancy. They may include:
Fatigue
Learning difficulties (the IQ (intelligence quotient )can be below 75)
Intellectual disability (possible, but does not get worse over time)
Muscle weakness
Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other
areas of the body
Difficulty with motor skills (running, hopping, jumping)
Frequent falls
Trouble getting up from a lying position or climbing stairs
Weakness quickly gets worse
Progressive difficulty walking
Ability to walk may be lost by age 12, and the child will have to use a wheelchair
Breathing difficulties and heart disease usually start by age 20
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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