Unmet needs for SMA

Spinal Muscular Atrophy (SMA) is a rare, and often fatal, genetic disorder that affects approximately 1 in every 10,000 births. This disease is due to defects in the SMN1 gene that produces a protein important for the survival and function of lower motor neurons. Deterioration and loss of lower motor neurons that innervate skeletal muscle leads to significant muscle atrophy. Muscle weakness is the most common and prominent feature of SMA, leaving many patients suffering from difficulty in performing motor tasks, such as sitting, standing, walking, breathing, and eating.

While there has been meaningful progress in the research of therapeutics aimed at slowing the loss of motor neurons, there continues to be a need for therapeutics that directly address muscle atrophy. Directly targeting the weakening of skeletal muscle may lead to improvements in muscle strength and motor function.

Patient Organizations

The organizations below provide additional information on SMA and resources available for patients and their families:

   SMA Foundation
 
 
   Cure SMA

SRK-015 is an investigational drug candidate. The effectiveness and safety of SRK-015 has not been established and SRK-015 has not been approved by the FDA or any other regulatory agency.
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