Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older
Basel, 26 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi ™ (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease . SMA causes progressive muscle weakness and atrophy, and significant unmet need remains, particularly in adults living with this condition. The CHMP recommendation was completed under the accelerated assessment pathway, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation. A final decision regarding approval is expected from the European Commission in the next two months and will be applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Evrysdi was approved by the U.S. Food and Drug Administration (FDA) in August 2020.“Our close partnership with the SMA community has enabled the development of the first ‘at-home’ treatment for SMA in infants, children and adults with varying levels of disease severity, the majority of whom remain untreated,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Office r and Head of Global Product Development....
Hirayama disease is a rare neuromuscular disease, which classically presents as lower motor neuron weakness and atrophy in the upper limbs and specifically the C7–T1 myotomes. Proposed pathogenesis relates to microcirculatory dysfunction in the territory of the anterior spinal artery caused by epidural venous plexus engorgement with forward displacement of the posterior dura and spinal cord during neck flexion, leading to chronic ischemic changes in the lower cervical anterior horn cells. Diagnosis hinges upon clinical and radiographic findings, and treatment is generally conservative given the self-limited nature of...
ConclusionThis allow us to make clear the very high attack rate and the great infective capacity of this B.1.1.7 variant of SARS-CoV-2.
ConclusionOur results indicate the importance of the DID test for the screening and monitoring of PCM and that the incidence ofP. lutzii might be greater than expected in areas where it is not the predominant PCM species. Therefore, this article may contribute to improving the knowledge and clinical management about this disease.
Russian media reports emergency officials saying 52 miners and rescuers have died in the Listvyazhnaya mine #emergencyofficials #coalmineexplosion #miners #rescuers #siberia
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Mayors across Brazil are divided on whether to maintain end-of-year festivities.
ConclusionWe formulated a method of predicting the logarithm posterior probability using two FDG models. We validated that the method can detect abnormal findings in CXRs and BCTs with both an acceptable performance for testing and a comparatively light workload for training.
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