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Spinal Muscular Atrophy: Late-July Monthly Roundup

by Nisha Cooch, PhD | Jul 31, 2019

In July, the literature pertaining to spinal muscular atrophy (SMA) has focused mainly on understanding the disease. Information on treatments has been published largely in review form, while there has also been some new information on managing the disease and what...

Spinal Muscular Atrophy: Late-July Monthly Roundup

by Nisha Cooch, PhD | Jul 31, 2019

In July, the literature pertaining to spinal muscular atrophy (SMA) has focused mainly on understanding the disease. Information on treatments has been published largely in review form, while there has also been some new information on managing the disease and what...

Spinal Muscular Atrophy: June Bi-Monthly Roundup

by Nisha Cooch, PhD | Jun 18, 2019

Since our last roundup, there has been continued focus in the spinal muscular atrophy (SMA) literature how best to treat the disease. There also continues to be research geared toward understanding the biology of the disease, as well as on SMA management, what it is...

Spinal Muscular Atrophy: Monthly Roundup

by Nisha Cooch, PhD | Jun 3, 2019

Much of the most recent research regarding spinal muscular atrophy (SMA) has focused on understanding the mechanisms mediating the disease as well as the clinical value of different pharmaceutical interventions. There have been several reviews on the latter topic,...

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Administering Zolgensma

Zolgensma, or onasemnogene abeparvovec, is an adeno-associated virus vector-based gene therapy that was approved by the FDA in May 2019. ...Read more

Zolgensma: Caregiver Counseling

As Zolgensma was recently approved by the FDA – in May 2019 – there has been little caregiver education on the therapy and how ...Read more

How Salbutamol May Help Patients with Spinal Muscular Atrophy: New Insights

The vast majority of spinal muscular atrophy (SMA) cases are caused by a mutation in the survival motor neuron 1 (SMN1) gene, which leads ...Read more

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Skye, First Baby in Connecticut Treated with Zolgensma, ‘Healthier and Stronger’

Skye, a nine-month-old baby with spinal muscular atrophy (SMA) type 1, is reaching new milestones every week and is “the happiest ...Read more
AveXis Knew of ‘Manipulated’ Data in Zolgensma Application and Failed to Inform FDA, Agency Says

AveXis, the company that developed the gene therapy Zolgensma to treat spinal muscular atrophy (SMA), knew of “data manipulation ...Read more
Clinicians Describe Rare Case of SMA Associated with Epilepsy in Teen

Clinicians have described the case of an Iranian teenager with a rare type of spinal muscular atrophy (SMA) associated with progressive ...Read more

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Spinraza Dose #7

I go in for my seventh Spinraza injection tomorrow. Like I’ve said before, it’s just kind of become another part of my routine ...Read more
Risdiplam Shows Potential in Babies and Adults with SMA

Risdiplam, an investigational oral therapy, shows higher SMN protein levels in both pediatric and adult SMA patients, according to an ...Read more
Preparing for Injection Days

In Michael Morale’s personal YouTube channel smajourney51, he did a video this week about preparing for injection days. He does ...Read more
Strategies for Combatting Migraines and Fatigue

I used to get migraines all the time, but now I get them much less frequently. Still, with the recent storm and dramatic shifts in the ...Read more

Spinal Muscular Atrophy: Late-July Monthly Roundup

Spinal Muscular Atrophy: Late-July Monthly Roundup

Spinal Muscular Atrophy: June Bi-Monthly Roundup

Spinal Muscular Atrophy: Monthly Roundup

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Recent SMA Neurologist News

Neurology Related Forum Topics

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