Gene Therapy is Transforming Spinal Muscular Atrophy Treatment Industry

SMA (Spinal Muscular Atrophy) is a very rare genetic disease that causes muscle weakness and muscle atrophy over a long period of time. This condition can have a serious impact on a child’s motor skills, making it difficult for them to crawl, walk, sit-up, and control their head movements. In India, SMA affects about 1 in 10,000 babies born, and about 38 people are carriers. To effectively manage SMA, precautionary measures such as newborn screening for high-risk babies, pregnancy carrier screening, and antenatal screening should be taken. Antenatal screening is the medical treatment conducted on a pregnant woman.

According to a recent report published by Allied Market Research, the spinal muscular atrophy treatment market is exhibited to grow at a noteworthy CAGR during the forecast timeframe. The growth of the spinal muscular atrophy treatment market is driven by the increasing number of SMA cases and the approval of SMA gene therapy as a treatment for SMA.

Early detection of SMA (Spinal Muscular Atrophy)-

Pregnancy carrier screening and newborn screening play an important role in the management of SMA, providing early detection, and early treatment, and making life better for people with SMA and their families. These tests enable healthcare professionals to diagnose SMA early in the disease course, enabling effective treatment plans and care for patients and their families.

Primary prevention techniques like carrier screening can be used during pregnancy to test for SMA in a fetus. This can be done by taking blood samples from the pregnant woman or by taking samples from umbilical cords to obtain fetal DNA samples for testing. Pregnancy carrier screening can help parents in making informed decisions during pregnancy and in planning the necessary care for the baby if it is determined that the baby has SMA. While the newborn screening is a test used shortly after the birth of a baby to diagnose SMA in a baby. It typically involves extracting small amounts of blood from a baby's heel and testing the blood for the SMA gene mutation.

Gene therapy has developed a new treatment for SMA. SMN1 gene is a defective or malfunctioning gene that plays a role in the functioning of motor neurons. The new version of SMN1 delivers a functional SMN1 gene to children under the age of 2 in a once-in-a-lifetime infusion. SMN1 helps motor neurons produce more SMN proteins, which are essential for muscle function.

Gene therapy for spinal arthritis approved by the Food and Drug Administration-

The United States Food and Drug Administration (FDA) approved Zolgensma, a gene therapy treatment for spinal muscular atrophy in May 2019 for children aged 2 and under with all types and forms of SMA. If the child has a genetic diagnosis of SMA and is less than 2 years old, the professional authorities can conduct a medical examination and further testing to determine whether the child is eligible for the SMA gene therapy.

Thus, the development of SMA therapy has provided patients and their families with hope and wellbeing. The spinal muscular atrophy treatment market is growing as more gene therapy is available and new treatments are being developed. By focusing on research, collaboration, and better treatment for patients, innovations make sure that SMA patients can receive superior medical attention and treatment options.

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