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.
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