The study analyzed that the spinal muscular atrophy therapeutics pipeline comprises approximately 16 drug candidates, of which one drug candidate is in Phase III stage of development, three drug candidates are in Phase II stage of development, three drug candidates are in Phase I stage, while seven drug candidates are in Pre-Clinical stage and two drug candidates in the Discovery stage 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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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. Cytokinetics Inc. is developing a drug by the name CK-2127107, which is in the Phase II stage of development obtained from a synthetics source, for the treatment of spinal muscular atrophy. Kowa Company Ltd. is under the process of developing Sodium Valproate, which is under Phase III stage of development as an inhibitor of 4-aminobutyrate transaminase, using a synthetic source, for the treatment of spinal muscular atrophy.
Many pharmaceutical companies are involved in the development of drugs for the treatment of spinal muscular atrophy therapeutics, and have their products in different phases. Kowa company Ltd. has its drug candidate by the name, Sodium Valproate is in the Phase III stage of development, for the treatment of spinal muscular atrophy. F. Hoffmann-La Roche Ltd. has two drug candidates in the Phase II stage of development namely, Olesoxime and RG 7916. Both are being developed for the treatment of spinal muscular atrophy. Cytokinetics, Inc. is in the process of developing a small molecule for the treatment of the disease which acts as a Fast-Skeletal muscle troponin activator.
AveXis, Inc., Sanofi Genzyme, AndroScience Corporation, AurimMed Pharma Inc., Neurodyn Life Sciences Inc., RaNA Therapeutics, Inc., and Neurotune AG, are some of the other players developing drugs that are in the therapeutic pipeline for spinal muscular atrophy.
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