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April 6, 2020April 6, 2020

Lack of Muscle Cell Signaling Seen to Kill Motor Neurons in SMA-LED2

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Problems with the secretion of survival signals from muscle cells seems to be the dominant driver of motor neuron loss in spinal muscular atrophy lower extremity predominant 2 (SMA-LED2), ... Read more

April 3, 2020April 2, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

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First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most ... Read more

April 3, 2020April 3, 2020

First Patient Treated in Phase 2/3 Trial Assessing Higher Dose of Spinraza

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The first patient has been dosed in the Phase 2/3 DEVOTE trial assessing the safety, tolerability, and efficacy of a higher dose of Spinraza (nusinersen) in patients of all ... Read more

April 2, 2020April 2, 2020

MDA Hosting Online Q&A on COVID-19 Today

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The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people with neuromuscular diseases might protect themselves during the COVID-19 pandemic. ... Read more

April 2, 2020April 2, 2020

FDA Issues No Fines for Data Problems in Zolgensma’s Application

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The U.S. Food and Drug Administration has decided not to fine or otherwise sanction Novartis for manipulated data included in the application that led to Zolgensma‘s approval to treat spinal muscular ... Read more

April 1, 2020April 1, 2020

Motor Gains and Safety with Zolgensma’s IT Use Continue, STRONG Data Show

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Young children given a single injection of Zolgensma into the spinal canal continue to show gains in motor function and safety, new interim data from the STRONG trial in spinal muscular ... Read more

March 30, 2020March 30, 2020

CHMP Recommends Conditional Approval of Zolgensma for SMA in Europe

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The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP), has recommended conditional approval of Zolgensma (onasemnogene abeparvovec) for the treatment of patients with spinal muscular atrophy (SMA) ... Read more

March 27, 2020March 27, 2020

Zolgensma Shows Benefits and Durability Across Trials in Infants, Novartis Says

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A single infusion of Zolgensma (onasemnogene abeparvovec-xioi) continues to lead to meaningful and sustained benefits in children with spinal muscular atrophy (SMA) treated in infancy, according to data from several ... Read more

March 27, 2020March 26, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

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The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. ... Read more

March 25, 2020March 25, 2020

Adult SMA Patients Benefit from Spinraza Treatment, Small Study Suggests

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Treatment of adult spinal muscular atrophy (SMA) patients with Spinraza (nusinersen, by Biogen) seems to be beneficial, despite the challenges associated with its administration, a small study suggests. Patients ... Read more

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