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December 6, 2019December 6, 2019

Regular CSF Exams Urged Following Spinraza Treatment to Screen for Injection Side Effects, Study Suggests

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The work-up of cerebrospinal fluid (CSF) samples from spinal muscular atrophy (SMA) patients treated with Spinraza (Nusinersen) revealed mild increases in protein content, which was attributed to lumbar punctures ... Read more

December 5, 2019December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 4, 2019December 4, 2019

Spinraza Stabilizes or Improves Motor Function in Older SMA Patients, US Real-world Study Suggests

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Spinraza (nusinersen) safely stabilizes or improves motor function in older patients with spinal muscular atrophy (SMA), according to a small U.S. real-world study. Researchers also pointed out that objective measures ... Read more

December 2, 2019December 2, 2019

Multidisciplinary Approach Improves Spinraza Dosing Process in SMA Patients, US Study Shows

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Adopting a multidisciplinary approach can optimize pretreatment evaluations and improve the dosing process for Spinraza (nusinersen) in people with spinal muscular atrophy (SMA), a U.S. study shows. This approach, which also used ... Read more

November 27, 2019November 27, 2019

Experts Barry Byrne, Jerry Mendell Lead NORD Webinar on Gene Therapy

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A glance around the walls of Barry J. Byrne’s office reveals a lot about the pediatric cardiologist who runs the Powell Gene Therapy Center at University of Florida (UF). In ... Read more

November 25, 2019November 26, 2019

Risdiplam, Potential Oral Therapy for All SMA Types, Granted Priority FDA Review

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The U.S. Food and Drug Administration (FDA) accepted Genentech‘s application requesting approval for risdiplam, its potential oral treatment for all types of spinal muscular atrophy (SMA), and gave it priority ... Read more

November 25, 2019November 25, 2019

MVICT May Be Better, More Reliable Test Than 6MWT in Trials of Ambulant SMA Patients, Study Finds

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Maximal voluntary isometric contraction testing (MVICT) may be a better outcome measure than the six-minute walking test (6MWT) in clinical trials involving people with spinal muscular atrophy who are ... Read more

November 22, 2019November 26, 2019

Targeted Genetic Screening Useful in Diagnosing Patients with SMA-like Symptoms

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Targeted next generation sequencing (NGS) — a powerful genetics method that can identify mutations in several genes simultaneously — may help diagnose people with spinal muscular atrophy (SMA)-like symptoms ... Read more

November 20, 2019November 20, 2019

SRK-015 Increases Myostatin Growth Factor Levels Up to 100-fold in SMA Patients, Early Trial Data Shows

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SRK-015, an investigational treatment designed to improve muscle strength and motor function in people with spinal muscular atrophy (SMA), interacts with its intended target in a dose-dependent manner, increasing ... Read more

November 18, 2019November 18, 2019

ALS Biomarkers Not Useful in Assessing Neurodegeneration in SMA Type 3 Patients Receiving Spinraza, Study Suggests

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The levels of neurofilament heavy chain and tau — two proteins commonly used as biomarkers of nerve cell degeneration in several neurodegenerative disorders — may not be useful as ... Read more

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