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December 18, 2018December 19, 2018

Poland to Reimburse Cost of Spinraza to Treat SMA, Ending Lengthy Struggle

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Poland’s Ministry of Health has agreed to pay for Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA) regardless of age or disease severity — ending a year-long struggle by ... Read more

December 17, 2018January 7, 2019

Post-Risdiplam Blood Test Could Assess SMA Patients’ Body-wide Protein Levels

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An increased amount of SMN protein — crucial to the survival of motor neurons — in the blood could reflect similar increases in the brain, spinal cord, and peripheral ... Read more

December 14, 2018January 3, 2019

Cure SMA Campaign Promotes Early Diagnosis and Treatment

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The Cure SMA educational campaign is designed to equip parents and healthcare professionals with the tools needed to recognize motor delays in spinal muscular atrophy (SMA) patients early, allowing infants ... Read more

December 12, 2018January 7, 2019

Damage That Marks SMA Type 3 and 4 Linked to Atrophy in Neck Area of Spinal Cord, Study Reports

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Later-onset forms of spinal muscular atrophy (SMA) are linked to extensive atrophy in the cervical spinal cord, particularly in gray matter of the vertebrae, that may begin long before symptoms ... Read more

December 10, 2018January 7, 2019

SMA and ALS Linked via ‘Central Hub’ Molecule Crucial to Protein Production, Study Reports

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Researchers at Stanford University School of Medicine found that a molecule called Activating Signal Cointegrator 1 (ASC-1) acts “a central hub” for proteins involved in amyotrophic lateral sclerosis (ALS) and ... Read more

December 7, 2018January 7, 2019

Experienced, Multidisciplinary Teams Can Ensure Success of Spinraza Programs, Study Reports

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Treatment of spinal muscular atrophy (SMA) with Spinraza (nusinersen) through the spinal canal is feasible and leads to high patient compliance as long as it’s done under the supervision ... Read more

December 4, 2018January 8, 2019

Zolgensma (AVXS-101) Under Priority FDA Review as Possible Gene Therapy for SMA Type 1

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The U.S. Food and Drug Administration (FDA) has granted priority review to Novartis’ Biological License Application (BLA) asking that Zolgensma (onasemnogene abeparvovec-xxxx) — previously called AVXS-101 — be approved as a ... Read more

December 3, 2018January 7, 2019

Intrathecal Dosing of Spinraza Is Safe in Adults with SMA Type 2 or 3, Study Shows

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Administration of Spinraza (nusinersen) directly into the spinal canal is feasible and well-tolerated by adults with spinal muscular atrophy (SMA) type 2 or 3. Still, such treatment can be challenging for ... Read more

November 30, 2018December 6, 2018

Scholar Rock Announces Additional Preclinical Data Supporting SRK-015 as Potential SMA Treatment

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Scholar Rock has announced the publication of new preclinical data supporting the potential therapeutic value of its experimental candidate therapy, SRK-015, in improving muscle function and strength in patients with spinal ... Read more

November 28, 2018January 7, 2019

Robotic Arm Controlled by Electrical Impulses Can Help SMA Patients Perform Daily Tasks

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The use of a robotic arm controlled by electrical impulses in the arm muscles may increase the independence of paralyzed spinal muscular atrophy (SMA) patients, according to a recent ... Read more

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