Abstract: The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.

Abstract: The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.

Abstract: The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.

Abstract: The invention relates to a portable material inspection device, with a light source, a polarizer, and an analyzer. The light source emits light through the polarizer to a material and the light reaches the analyzer. The angle or position of the incident emitted light, either polarized or unpolarized is freely adjustable. The inspection device may have an waterproof and dust resistant external shell and be relatively small, less than 7 inches by 5 inches by 2 inches.

Abstract: The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.

Abstract: Methods and systems are provided for mitigating excessive exhaust pressures in an engine. In one example, a method may include adjusting an intake throttle responsive to exhaust pressure upstream of an exhaust turbine being higher than a threshold without reducing boost level. In this way, boost pressures may be maintained while reducing exhaust pressures.

Abstract: Methods and systems are provided for mitigating excessive exhaust pressures in an engine. In one example, a method may include adjusting an intake throttle responsive to exhaust pressure upstream of an exhaust turbine being higher than a threshold without reducing boost level. In this way, boost pressures may be maintained while reducing exhaust pressures.

Abstract: Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.

Abstract: Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.

Abstract: Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.

Abstract: Aryl substituted thiazol-2-yl-piperidines and related compounds useful as modulators of survival motor neuron (SMN) protein production are provided herein. Without being bound to any particular theory it is believed the aryl substituted thiazol-2-yl-piperidines and related compounds provided herein act to increase production of the SMN2 form of survival motor neuron protein. These compounds are useful for treating spinal muscular atrophy. Pharmaceutical compositions containing a carrier and one or more of the aryl substituted thiazol-2-yl-piperidine or related compounds described herein are also provided. Methods of treating spinal muscular atrophy are also provided by this disclosure.

Abstract: A metal channel for positioning and retaining metal studding and wall panels to form partition walls, comprising a central web with retaining flange portions along its opposite sides. The web is segmented, being divided along its length into discrete segments by transverse slots. One of the flange portions is similarly divided into segments by transverse slots aligned with the slots. Adjacent segments of the flange portions are however connected together by expansible bridges and optionally by frangible bridges. The other of the flange portions is provided with lines of perforation aligned with the slots and defining flexure zones. The channel is formed straight but can be bent into smooth curves by expansion of the expansible bridges.