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    Home > Biochemistry News > Biotechnology News > Health announces SMA data before Spinraza treats symptoms 96% of patients can walk with aids

    Health announces SMA data before Spinraza treats symptoms 96% of patients can walk with aids

    • Last Update: 2020-06-16
    • Source: Internet
    • Author: User
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    The latest results of the NURTURE study, released today, are the longest-running study to date for patients with spinal muscular dystrophy (SMA) symptoms and will change expectations for early treatment of Spinraza (nusinersen)The data showed that in infants diagnosed with SMA, early and continuous use of Spinraza treatment lasted 4.8 years, achieving an unprecedented survival rate (100%), and none of the patients needed permanent ventilationCompared to the natural course of the disease, patients continued to gain progressive gain in motor function, with 96% being able to walk with assistThe results were presented at the Virtual Cure SMA Research and Clinical Care Conference on June 10-12NURTURE is an ongoing global, open-label, single-group, efficacy and safety study of 25 SMA infant spandal patients who were diagnosed with SMA by genetic testing and given the first dose of Spinraza treatment within 6 weeks of birthThe patient is in the pre-symptom stage of the disease when the drug is first administeredThe study was extended by an additional three years to assess the long-term efficacy and safety of Spinraza treatment up to the age of 8, and to further understand the impact of early treatmentThe data included nearly a year of additional follow-up visits to PATIENTs in the NURTURE studyAs of February 2020, all patients receiving treatment (n-25; median age 3.8 years) were alive and without permanent ventilationIn the natural history of the disease, most SMA-type 1 children live less than 2 years without treatmentIn addition, all children who reached the milestone of being able to walk independently (many within the normal development altimency schedule) maintained this ability from the first milestone to the last visitAdditional results from the latest mid-term analysis as of February 2020 show that all patients who were previously able to walk under assisted (92%) and walk independently (88%) maintained this ability for 11 months after the last data cut off; - In an additional 11-month follow-up, one child was given the ability to walk under assisted mobility (increasing the proportion of patients walking with assisted walking to 96%), and the maximum score was reached on the infant neuromuscular disease test scale at Pennsylvania Children's Hospital, increasing the total number of patients with the highest score to 21 and the proportion to 84% (n?21/25); -- Patients carrying 2 copies of the SMN2 gene were able to get an unusual advance on the Hammersmith Functional Exercise Scale (HFMSE)Spinraza is well tolerated and no new safety issues were found during the extended follow-up periodNo children have discontinued their studies because of adverse events associated with treatmentThese results confirm that early and continuous treatment of Spinraza has a significant impact on SMA infants, with many patients experiencing consistent progress in motor function with children of similar age without SMAThese new results continue to reinforce the substantial benefits of timely diagnosis, early and long-term use of Spinraza treatmentSMA is a rare genetic neuromuscular disease characterized by the loss of motor neurons in the spinal cord and low brain stem, which can lead to severe developive muscle atrophy and weakness About one in every 10,000 live births is diagnosed with SMA, and patients of all ages are affected by the disease The disease is the number one genetic disease that causes infant death Spinraza is the first approved treatment for SMA infants, children and adults and has been approved in more than 50 countries As of March 31, 2020, more than 10,000 patients worldwide have received Spinraza treatment The drug is the only SMA drug for a wide range of patient groups with unparalleled real-world experience and a strong level of clinical evidence Source: New Results From Landmark Study Show Which Pre-Symptomatic SMA Patients Treated With SPINRAZA? (nusinersen) Continue to The Stagain Sustained Benefit From Treatment
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