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SMA in Late February: Roche’s Sunfish Trial, Intrathecal Nusinersen Treatment & More

by Nisha Cooch, PhD | Feb 26, 2020

Perhaps the biggest SMA news this month has been the success of Roche’s Sunfish trial, but fundamental research, such as those into the mechanisms of the disease and the implications of certain interventions, continue. Below is a roundup of the latest news and...

SMA in Late February: Roche’s Sunfish Trial, Intrathecal Nusinersen Treatment & More

by Nisha Cooch, PhD | Feb 26, 2020

Perhaps the biggest SMA news this month has been the success of Roche’s Sunfish trial, but fundamental research, such as those into the mechanisms of the disease and the implications of certain interventions, continue. Below is a roundup of the latest news and...

SMA HCP Roundup, mid-February: X-linked infantile SMA, Risdiplam News & More

by Nisha Cooch, PhD | Feb 18, 2020

Research on SMA continues to forge ahead in 2020, with new results in from Roche’s latest investigation into its oral SMA drug, risdiplam. New tools for screening are being revealed as legislative changes are pushing more and more toward requiring infants born in the...

Rolling From Side To Side – Focusing On Abductor & Adductor Muscles

by Emily Jones | Feb 10, 2020

Patients with SMA often spend the majority of their time either in their wheelchair or in their bed. Since they can be fairly limited to these two places, it is important to increase their independence within those areas. Although my patient with SMA, Michael, will...

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Risdiplam Succeeds in Treating Type 1 Babies with FIREFISH Meeting Main Goal, Genentech Says

Genentech announced, without providing details, positive topline results from the pivotal second part of its FIREFISH clinical trial of […]
Adding Cancer Medicine to Spinraza Boosts SMN Production, Early Study Finds

Combining Spinraza with low-dose panobinostat — the active substance in an approved treatment for a blood cancer — may […]
Adults With SMA Tend to Report Low Prevalence of Non-Motor Symptoms, Study Finds

Adults with spinal muscular atrophy (SMA) type 2 and 3 tend to report a low prevalence of non-motor symptoms, a study finds. However, some […]

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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 […]
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 […]
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 […]
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 […]

SMA in Late February: Roche’s Sunfish Trial, Intrathecal Nusinersen Treatment & More

SMA in Late February: Roche’s Sunfish Trial, Intrathecal Nusinersen Treatment & More

SMA HCP Roundup, mid-February: X-linked infantile SMA, Risdiplam News & More

Rolling From Side To Side – Focusing On Abductor & Adductor Muscles

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