News

August 17, 2017August 17, 2017

AAN Forms Therapy Pricing Panel to Help Neurologists Treat SMA and Other Diseases

As potentially lifesaving, but costly, therapies become available for rare genetic diseases — such as Spinraza (nusinersen) for spinal muscular atrophy (SMA) — neurologists are finding themselves in the ... Read more

August 15, 2017August 15, 2017

Roche Begins Enrolling SMA Type 1 Infants in Phase 2 Trial of Therapy Targeting SMN2

News

Roche has started enrolling infants with spinal muscular atrophy (SMA) type 1 in a Phase 2 trial of its SMN2-targeting therapy RO7034067, following positive early results from a similar ... Read more

August 10, 2017

Texas is Latest State to Consider Requiring Newborn Screening for SMA

News

Texas is considering adding spinal muscular atrophy (SMA) to its list for newborn screening in the wake of the U.S. Food and Drug Administration’s approval in December of Spinraza (nusinersen), the ... Read more

August 8, 2017

Designer Partners with 3D Printer to Build Custom Exoskeleton for Kids with SMA

News

A designer trying to manufacture a custom-fitting exoskeleton for children with spinal muscular atrophy (SMA) has partnered with the 3D printing company Sinterit. According to a 3D Printing Industry news ... Read more

August 3, 2017

SMA Awareness Month’s Goal: Newborn Screening in Every State

News

With August recognized as SMA Awareness Month, this year the spinal muscular atrophy (SMA) community is focused on promoting nationwide newborn screening. Since 1996, Cure SMA has coordinated efforts to ... Read more

August 1, 2017

New ASO Compounds Show Potential as Systemic Therapy for SMA, Study Shows

News

Subcutaneous administration of a new class of antisense oligonucleotide (ASO) compounds called tricyclo-DNA may be an efficient therapy for spinal muscular atrophy (SMA), according to a study in mice. ... Read more

July 28, 2017July 31, 2017

Biogen’s Spinraza Surpassing Expectations as More Insurers Cover It or Relax Coverage Restrictions

News

Biogen’s Spinraza (nusinersen), an expensive but lifesaving medication for thousands of babies born with spinal muscular atrophy (SMA), is becoming available to more and more Americans as private and government ... Read more

July 27, 2017July 27, 2017

Salbutamol Improves Respiratory Muscle Strength of Children With SMA, Study Reports

News

A year’s worth of salbutamol improved the breathing function and respiratory muscle strength of children with spinal muscular atrophy, according to a small-sample study. The research, “Effect of Salbutamol ... Read more

July 25, 2017July 26, 2017

Education Is Essential to Success of SMA Carrier Screening Programs, U.K. Study Finds

News

Families living with spinal muscular atrophy (SMA), like most people, favor screening for genetic disorders. Yet those who lack experience with SMA generally take a far more negative view ... Read more

July 20, 2017July 26, 2017

Quest Diagnostics’ New Genetic Carrier Screening Test, QHerit, Includes SMA

News

Quest Diagnostics has launched a new screening test to assess the possibility that parents carry genes that might cause spinal muscular atrophy and a range of other diseases in ... Read more