Spinal muscular atrophy or SMA is a genetic disorder that affects the nervous system, impairing voluntary muscle movement. It is a hereditary autosomal recessive disease and causes mortality in children. Earlier onset of SMA makes the impact greater, damaging the motor function of the child to a larger extent. Children who show symptoms at birth or infancy have type 1 SMA, which leads to minimal motor skill functioning. However, the ones who develop it as children contract type 2 and 3 global SMA market and the ones who get it in teenage or as adults have type 4 SMA. These latter patients have a slightly better ability to maintain their motor skills than the type 1 cases.

Deficiency of a motor neuron protein called survival motor neuron (SMN) leads to SMA. This protein is extremely important for normal motor neuron function. The deficiency of this protein is caused due to mutation of chromosome 5 inside a gene called SMN1. This deficiency is partly compensated by the neighboring SMN2 genes.

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Orphanet, a journal of rare diseases, states that out of 6,000 births, one infant suffers from SMA. The statistics also indicate that there are more than 10 million people in the U.S. carrying the SMA gene. About 60% of infants who are born with type 1 SMA do not survive beyond 2 years. However, the ones with type 2 SMA have a moderate impact on their lives and the ones with type 3 SMA and type 4 SMA live out a normal lifespan.

The global spinal muscular atrophy market is segmented into products that are still in the pipeline. The key product in the phase 3 for the late stage candidates segment is ISIS-SMN Rx. On the other hand, products in the phase 1 and 2 for early stage candidates are LMI070, Olesoxime (TRO19622), RO6885247, RG3039, CK-2127107, and CB.SMN. So far, no definitive cure has been devised for spinal muscular atrophy.

Regionally, the global spinal muscular atrophy market has a strong opportunity for growth in regions such as Europe and the United States, as a high number of SMA cases are diagnosed in these regions. In addition to this, the research and development on drugs for SMA might pick up pace with private organizations and governments in these regions making serious attempts at creating awareness about the disease.

The greater number of carriers in this region will also be the reason for faster growth of this market in the next few years. The incentive schemes for developing therapeutic alternatives for rare diseases will also contribute to the success of the SMA market in these regions.

Meanwhile, companies are making ardent efforts to come up with solutions for SMA, a disease that can only be managed with physiotherapy as of now. As the disease worsens, medical practitioners also often have to treat patients for respiratory and gastrointestinal disorders, which are commonly seen in SMA patients.

Companies such as Novartis AG, AveXis, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., and Isis Pharmaceuticals, Inc. are making serious efforts at designing solutions to make SMA management a lot easier for both the patient and the caregiver.

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