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September 29, 2020September 30, 2020

Infants Receiving Evrysdi Continue to Improve and Achieve Motor Milestones, Phase 2/3 Trial Data Show

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Infants with spinal muscular atrophy (SMA) type 1 receiving the therapeutic dose of Evrysdi (risdiplam) continue to improve and achieve motor milestones, according to two-year data from the first ... Read more

September 28, 2020September 28, 2020

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

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It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they ... Read more

September 24, 2020September 24, 2020

Novartis to Open, at FDA Request, New Trial of Zolgensma for Older Patients

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Novartis will open a new clinical trial investigating Zolgensma, given via an injection directly into the spinal canal, after the U.S. Food and Drug Administration (FDA) recommended a “pivotal confirmatory ... Read more

September 23, 2020September 23, 2020

Spinraza Leads to Motor Gains Over Time in SMA Type 3 Adults, Real-Life Study Finds

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About one year of treatment with Spinraza (nusinersen) gradually and significantly improves motor function in adults with spinal muscular atrophy (SMA) type 3 — especially those unable to walk ... Read more

September 21, 2020September 21, 2020

Evrysdi Open to Eligible Patients in UK Through Early Access Program

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Evrysdi (risdiplam), Roche’s oral therapy for spinal muscular atrophy (SMA), will be available to select patients in the U.K. before its potential regulatory approval through an Early Access to ... Read more

September 18, 2020September 18, 2020

GridPad Trilogy Communication Device Aids People With Speech and Motor Disorders

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Control Bionics has announced the launch of the GridPad Trilogy, an augmentative and alternative communication (AAC) device intended to aid people living with a range of conditions that affect movement ... Read more

September 16, 2020September 16, 2020

Electric Bike Technologies Donates Liberty Trikes to Children to Aid Mobility

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To help children with spinal muscular atrophy (SMA) gain mobility and independence, Electric Bike Technologies (EBT) is donating 10 Liberty Trikes — and plans to double that gift next year. ... Read more

September 14, 2020September 14, 2020

AveXis Now Known as Novartis Gene Therapies, Focus of Continuing Work

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Two years after acquiring the gene therapy company AveXis, Novartis has renamed it Novartis Gene Therapies to underscore the potential value of developing such treatments for genetic diseases. This ... Read more

September 11, 2020September 11, 2020

NICE Widens Its Zolgensma Appraisal Due to European Marketing Authorization

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England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy (SMA), according to a press release from SMA ... Read more

September 9, 2020September 9, 2020

Activities Underway for Newborn Screening Awareness Month

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As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. ... Read more

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