Spinal Muscular Atrophy Therapeutics Pipeline Analysis 2017

The study analyzed that the therapeutics pipeline comprises approximately 16 drug candidates in different stages of development. Spinal muscular atrophy is defined as the inherited genetic disease that is characterized by a failure of nerve cells called motor neurons. Motor neurons are responsible to accept the nerve impulses transmitted from the brain to the spinal cord and transmit the impulses to the muscle with the help of peripheral nerves. The loss of motor neurons leads to muscle weakness in muscles that are closest to the trunk of the body such as back, hips and shoulders.

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Imago Pharmaceuticals, Inc. is using nucleic acid therapeutics technology platform for the treatment of spinal muscular atrophy. Nucleic acid therapeutics technology includes large and innovative class of drugs that can modulate the function of target ribonucleic acid, or RNA, to ultimately affect the production of disease-associated proteins. Nucleic acid therapeutics comprise of complex mixtures of various chemical entities known as stereoisomers. Some stereoisomers in these mixtures have therapeutic effects, while others are less beneficial or contribute to undesirable side effects. Uncontrolled stereoisomer drug mixtures can lead to suboptimal efficacy and increased risk and safety concerns.

The research also found that there are several companies that use synthetic sources for the development of drugs for the treatment of spinal muscular atrophy.

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Some of the other key players developing drugs for the treatment of spinal muscular atrophy include Cytokinetics, Inc., F. Hoffman La-Roche Ltd, WAVE Life Sciences Ltd. and others.