Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disorder for which there is no cure. It is a result of a missing or mutated gene and affects the voluntary muscles throughout the body. This disease affects 1 in 6000 babies born and about 1 in 40 people are genetic carriers. It is one of the most prevalent genetic disorders and can affect individuals of any age, race or gender.
Individuals with SMA have difficulty with activities such as crawling, walking, head and neck control, and swallowing. They have weakness in limbs, with legs generally more affected than arms, and difficulty feeding and swallowing. Patients also have an increased risk of developing pneumonia and other lung problems due to involvement of respiratory muscles. Sensation is not affected and intellectual activity is normal.
SMA is an autosomal recessive genetic disease. This means that in order for a child to be affected, both parents must be carriers of the abnormal gene and both must pass this gene onto their child. If both parents are carriers, there is a 25% chance of the child inheriting the disease.
The missing or mutated gene is SMA is called survival motor neuron 1 (SMN1), which produces a protein called Survival Motor Neuron (SMN) protein. This protein is critical to the survival and health of motor neurons, which are nerve cells in the spinal cord that send out nerve fibers to muscles throughout the body. Without SMN, nerve cells atrophy and eventually die, resulting in muscle weakness.
The list of signs and symptoms mentioned in various sources for Spinal Muscular Atrophy includes the symptoms listed below:
. Muscle weakness
. Muscle atrophy
. Leg weakness
. Lack of muscle tone
. Thin muscle mass
. Absent tendon stretch reflexes
. Respiratory distress
. Feeding difficulty
. Congenital contractures
. Flaccid infants
. Lack of infant movement
. Lack of infant head control
. Protrusion of tongue when crying
. Difficulty walking
. Difficulty crawling
. Difficulty sitting up
. Involuntary facial muscle twitching
. Muscle twitching
. Atrophic muscle changes
. Reduced sexual potency
. Swallowing difficulty
. Absent pyramidal sensory sign
. Absent pyramidal cerebellar signs
. Enlarged male breasts
. Distal sensory neuropathy
. Male infertility
. Large calf muscles
Stem Cell Treatment for Spinal Muscular Atrophy/ Stem cell treatment for SMA
Stem cell treatment may cure symptoms of SMA and we use adult stem cells derived from umbilical cord (umbilical cord blood stem cells and umbilical mesenchymal stem cells). This type of stem cell treatment offers remission in 85% of patients, as well as helping to diminish the symptoms of neurogenic dystrophy in patients in 90% of cases.*
Stem cell therapy to treat Spinal Muscular Atrophy focuses on completely replacing motor neurons that have been damaged or destroyed due to the disease process. Not only will such stem cell treatments slow down the disease progression, but may also be used to treat a variety of disorders including spinal cord injuries or other neuromuscular damage or disease processes that interrupts the flow of information signals controlling nerve and muscular movement.
Stem cell therapies and technologies offer hope now for enhancing quality of life, restoring mobility and muscular function as well as range of motion and stability for individuals currently diagnosed with SMA.
For more information on stem cell treatment for SMA, please complete a medical form