Considerations for Treatment in Clinical Care of Spinal Muscular Atrophy Patients
Spinal Muscular Atrophy is a neurodegenerative disease which can lead to muscle weakness, paralysis, and in some cases death. There are many factors that contribute to the severity of symptoms and those factors can be used to determine the best course of
Stephanie Voight, Kapil Arya
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The distal form of spinal muscular atrophy: an unusual case demonstrating the intermediate variety [PDF]
David Isenberg, Pauline A Kahn
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An integrated computational approach evaluating the binding between pre‐let‐7 microRNA and small‐molecule kinase inhibitors (SMKIs) revealed varied pre‐let‐7 binding affinities among the advanced SMKIs. The results justified the characterization of potential RNA targets for kinase inhibitors and small molecules with protein targets in general.
Soma Roy, Yang Liu, Peng Wu
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Background Spinal Muscular Atrophy (SMA) is a rare autosomal recessive genetic disorder characterized by degeneration of motor neurons in the spinal cord, resulting in progressive limb muscle weakness, atrophy, and severe scoliosis.
Zhuangyuan Chen +5 more
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Hereditary distal muscular atrophy with vocal cord paralysis and sensorineural hearing loss: a dominant form of spinal muscular atrophy? [PDF]
Eugen Boltshauser +3 more
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Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks
A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan +8 more
wiley +1 more source
Cell adhesion molecule N-CAM is expressed by denervated myofibres in Werdnig-Hoffman and Kugelberg-Welander type spinal muscular atrophies. [PDF]
F S Walsh, S.E. Moore, B D Lake
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Pigment‐Macromolecule Complexes Isolation from Sea Urchin Biomineral Waste for Coloring Materials
A methodology has been developed to extract the colored organic fraction occluded within seashell biomineral waste. Applied to sea urchin skeletons, free polyhydroxylated naphthoquinone (PHNQ) pigments and PHNQ‐macromolecule complexes are isolated. These complexes show superior pH stability in purple hues compared to free PHNQ, which shifts from red to
Claudio Ferreira +12 more
wiley +1 more source
PROGRESSIVE SPINAL MUSCULAR ATROPHIES. (International Review of Child Neurology Series). Edited by Ingrid Gamstorp and Harvey B. Sarnat. Published by Raven Press. $28.00 Cdn. approx. [PDF]
John A.R. Tibbies
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Several regulatory initiatives have been made to clarify the acceptability and requirements of real‐world data and real‐world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the ...
Suguru Okami +2 more
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