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December 10, 2018December 10, 2018

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, 2018December 10, 2018

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, 2018December 4, 2018

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, 2018December 5, 2018

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, 2018December 4, 2018

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

November 26, 2018November 29, 2018

Wisconsin Mom Credits Spinraza with Restoring ‘Quality of Life’ to Her Kids with Type 2 and 3 Disease

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Danyelle Sun, a Milwaukee mother of two children with spinal muscular atrophy (SMA), says Biogen’s Spinraza (nusinersen) has rescued both her kids from potentially lifelong disabilities. Sun, 35, is the ... Read more

November 21, 2018November 28, 2018

Scientists Re-create All Human SMA Types, Including Type 4, in Fruit Fly Models

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Researchers from the University of North Carolina have described 14 different fruit fly models that mimic all the types of spinal muscular atrophy (SMA) seen in patients. These include SMA ... Read more

November 19, 2018November 26, 2018

Researchers Develop Blood Test to Screen Newborns for SMA and Identify Carriers

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Researchers have developed a rapid, specific blood test to screen newborns for spinal muscular atrophy (SMA) and identify carriers — those who do not have the disease but can pass ... Read more

November 16, 2018November 20, 2018

LMI070 Oral Candidate For SMA Type 1 Holds Promise, Preclinical Study Shows

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LMI070 (branaplam), a candidate oral therapy under development by Novartis, is being tested in a Phase 1/2 clinical trial for infants with spinal muscular atrophy (SMA) type 1. A recent study ... Read more

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