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

Immediate Treatment Advised for Newborns with 4 Copies of SMN2 in Updated Guidelines

Feb 14, 2020 07:00 am | Ana Pena, PhD

 

SMA treatment guidelinesNewborns diagnosed with spinal muscular atrophy (SMA) via newborn screening (NBS) and who carry four copies of the SMN2 gene should start treatment immediately, just as those with two or three SMN2 copies should, according to updated guidelines by a working group of SMA experts. The group also emphasized new trial data supporting the importance […]

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18 States in US Screening Newborns for SMA as Efforts Continue for More, MDA Says

Feb 12, 2020 07:00 am | Grace Frank

 

Pompe/SMA newborn screeningEighteen states are now screening newborns for spinal muscular atrophy  (SMA), the most recent neuromuscular disease added to a list of serious genetic disorders that infants can be tested for shortly after birth, advocacy officers at the Muscular Dystrophy Association (MDA) said. California, the most populous state with about 39 million people, also is in the […]

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FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

Feb 11, 2020 11:51 am | Hawken miller

 

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to provide feedback on therapy development, set regulatory priorities, and contribute to patient-reported outcomes. Starting […]

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Targeting Defects in Cell’s ‘Skeleton’ May Restore Cellular Function in SMA, Early Study Shows

Feb 10, 2020 10:30 am | Ana Pena, PhD

 

cell functionLow levels of the survival motor neuron (SMN) protein — the major root cause of spinal muscular atrophy (SMA) — may lead to defects in the “skeleton” of cells,  which can affect cell growth and function, an early study in yeast suggests. In particular, one of the defects described in the study is the excessive […]

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