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January 20, 2020January 20, 2020

CSF Protein Profile May Help Predict Spinraza Responses in Late-onset SMA Patients, Study Shows

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Analysis of the protein levels in the cerebrospinal fluid (CSF) of people with late-onset spinal muscular atrophy (SMA) may help to identify biomarkers that not only monitor but also predict ... Read more

January 17, 2020January 17, 2020

Magnetic Rods May Be Alternative to Correct Scoliosis in Children With SMA, Study Suggests

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Spine surgery using magnetically controlled growing rods (MCGR) may be an option to correct spinal deformity, or scoliosis, in young children with spinal muscular atrophy (SMA) and avoid the ... Read more

January 15, 2020January 16, 2020

Roche Starts Global Early Access Program for SMA Therapy Risdiplam in Europe

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Roche has kicked off a worldwide early access program for its experimental therapy risdiplam by making it available to Europeans with the most serious forms of spinal muscular atrophy ... Read more

January 13, 2020January 13, 2020

Fetal Gene Therapy May Ease SMA Symptoms, Prolong Survival, Mouse Study Shows

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Gene therapy administered while newborns are still in the womb may lessen spinal muscular atrophy (SMA) symptoms and prolong survival, a mouse study shows. The study, “Fetal Gene Therapy ... Read more

January 10, 2020January 10, 2020

NORD Webinar Features SMA Mom Discussing Zolgensma Gene Therapy

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Just 27 days after Nicole Almeida gave birth to her son, Matteo, the baby received a one-time infusion of what one day would be known as Zolgensma. That made ... Read more

January 8, 2020January 8, 2020

ZPR1 Protein May Be New Potential Therapeutic Target for SMA, Preclinical Study Suggests

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Increasing the amount of ZPR1 — a protein found in low levels in people with spinal muscular atrophy (SMA) — improved motor function and extended the lifespan of a ... Read more

January 6, 2020January 6, 2020

Protest Secures Spinraza for All SMA Patients in Romania

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An October 2019 protest by adults with spinal muscular atrophy led to an agreement under which all of Romania’s 152 known SMA patients — and those yet to be diagnosed ... Read more

January 3, 2020January 4, 2020

Up to 100 Free Zolgensma Treatments Going Out Worldwide in 2020 Under Access Program

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Novartis plans to give up to 100 doses annually of Zolgensma, its costly gene therapy for spinal muscular atrophy (SMA), free-of-charge to eligible young children outside the U.S. with this progressive, neuromuscular ... Read more

December 18, 2019December 20, 2019

Parents of 2 Babies Treated with Zolgensma in July: We Made the Right Choice

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The parents of two babies treated in July with Zolgensma, the first gene therapy approved for spinal muscular atrophy or any neuromuscular disease, are confident they made the right choice ... Read more

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CSF Protein Profile May Help Predict Spinraza Responses in Late-onset SMA Patients, Study Shows

Magnetic Rods May Be Alternative to Correct Scoliosis in Children With SMA, Study Suggests

Roche Starts Global Early Access Program for SMA Therapy Risdiplam in Europe

Fetal Gene Therapy May Ease SMA Symptoms, Prolong Survival, Mouse Study Shows

NORD Webinar Features SMA Mom Discussing Zolgensma Gene Therapy

ZPR1 Protein May Be New Potential Therapeutic Target for SMA, Preclinical Study Suggests

Protest Secures Spinraza for All SMA Patients in Romania

Top 10 SMA Stories of 2019

Up to 100 Free Zolgensma Treatments Going Out Worldwide in 2020 Under Access Program

Parents of 2 Babies Treated with Zolgensma in July: We Made the Right Choice

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