Spinal Muscular Atrophy (SMA) Treatment in Children

Spinal Muscular Atrophy (SMA) Treatment in Children

Spinal Muscular Atrophy (SMA) is a rare but serious genetic disorder that affects the motor nerve cells in the spinal cord, leading to progressive muscle wasting and weakness. Early diagnosis and intervention are crucial to managing the condition effectively and improving the quality of life in children affected by this disease. At our medical center, we provide comprehensive care and support for children diagnosed with spinal muscular atrophy.

What Are SMA Disorders?

SSpinal muscular atrophy is a hereditary condition caused by a deficiency of a motor neuron protein called SMN (survival motor neuron), which is essential for the proper functioning of motor neurons. Without this protein, nerve cells lose the ability to communicate with muscles, resulting in muscle atrophy and loss of movement over time. Spinal Muscular Atrophy (SMA) Treatment for Children in Dubai focuses on early diagnosis and advanced interventions to manage symptoms and improve quality of life.

Types of SMA Disorders in Children

There are several types of spinal muscular atrophy, classified based on the age of onset and severity of symptoms

SMA Type 1 (Werdnig-Hoffmann Disease)

This is the most severe and common form, appearing in infants before 6 months of age. Children with spinal muscular atrophy type 1 typically have difficulty breathing, swallowing, and moving.

SMA Type 2

Usually begins between 6 and 18 months. Children may sit but typically cannot stand or walk unaided.

SMA Type 3 (Kugelberg-Welander Disease)

Milder form with symptoms starting after 18 months. Individuals can usually walk but may have difficulty with stairs and balance.

SMA Type 4

Adult-onset form that usually begins in the second or third decade of life with mild motor impairment.

Spinal Muscular Atrophy Symptoms

The symptoms of spinal muscular atrophy can vary depending on the type and severity of the condition. Common spinal muscular atrophy symptoms include

Muscle weakness and decreased muscle tone

Limited mobility or inability to sit, stand, or walk independently

Breathing and swallowing difficulties

Poor head control in infants

Fasciculations (muscle twitching), especially in the tongue

Early signs in infants, particularly with spinal muscular atrophy type 1, include floppy limbs, difficulty lifting the head, and feeding challenges.

Diagnosis and Treatments

Diagnosing SMA involves a combination of clinical evaluation and specialized tests, including

Genetic testing to confirm the presence of SMN1 gene mutation

Electromyography (EMG) and nerve conduction studies

Muscle biopsy in rare cases

Treatment

While there is currently no cure for SMA, Spinal Muscular Atrophy (SMA) Treatment for Children in Dubai offers several options that can significantly improve outcomes and enhance quality of life.

Gene therapy (e.g., Zolgensma):

A one-time treatment that replaces the faulty SMN1 gene with a functional copy.

SMN-enhancing drugs (e.g., Spinraza, Evrysdi)

These medications increase the production of the SMN protein from the SMN2 gene.

Supportive therapies

Physical and occupational therapy, respiratory support, nutritional care, and orthopedic interventions play a critical role in managing SMA symptoms

Dr. Vivek Mundada, a leading pediatric neurologist in Dubai, leads a multidisciplinary team that works closely with families to provide personalized care plans for children with spinal muscular atrophy, focusing on improving motor function, maintaining independence, and supporting emotional well-being.