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See below for a daily digest of the latest SMA news!

Zolgensma Approved For SMA Treatment in Japan

Mar 23, 2020 07:00 am | Margarida Azevedo, MSc

 

Zolgensma approvalThe Japanese Ministry of Health, Labour and Welfare approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of spinal muscular atrophy (SMA) patients under the age of two, Novartis announced. The approval includes toddlers and newborns who are pre-symptomatic at diagnosis, meaning those who have not yet shown symptoms. Eligible patients for treatment must not show elevated antibodies against […]

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Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Mar 20, 2020 11:10 am | Larry Luxner

 

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) — launched in 2011 by the European Union […]

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FDA Grants Shift’s E1v1.11 Orphan Drug Status for SMA

Mar 20, 2020 08:30 am | Margarida Azevedo, MSc

 

E1v1.11Shift Pharmaceuticals said its investigational lead therapy E1v1.11 has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) as a potential treatment option for spinal muscular atrophy (SMA). SMA is caused by mutations in the survival motor neuron 1 (SMN1) gene, which leads to lower production of SMN, a key protein for motor neuron […]

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Study Addresses Dilemmas Regarding Newborn Screening and SMA Treatment

Mar 18, 2020 07:30 am | Marisa Wexler, MS

 

newborn screeningAs newborn screening for spinal muscular atrophy (SMA) becomes more common, ethical dilemmas will arise about the best treatment strategy for individuals with four copies of the SMN2 gene, a new study contends. The study, “Infants Diagnosed with Spinal Muscular Atrophy and 4 SMN2 Copies through Newborn Screening – Opportunity or Burden?” was published in […]

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