Spinal Muscular Atrophy (SMA) Symptoms, Diagnosis, Prognosis, and Treatment News Today

April 20, 2018April 19, 2018
The Never-ending Quest to Gain Weight
Columns, Life, One Cup at a Time - a Column by Alyssa Silva
Although Mother Nature seems to have other plans lately, the days crossed off our calendars have us inching closer ... Read more
April 20, 2018April 20, 2018
#AAN2018 — Spinraza Improves Motor and Respiratory Function in SMA Children, Trials Show
News
Spinraza (nusinersen) improves motor and respiratory function in children with spinal muscular atrophy (SMA), according to results from ... Read more
April 18, 2018
#AAN2018: SMA Treatment Candidate RG7916 Increases Amount of Key SMN Protein, Clinical Trials Show
News
Roche’s treatment candidate RG7916 increased the amount of full-length, functional SMN protein in spinal muscular atrophy (SMA) ... Read more
April 18, 2018April 17, 2018
A Message for Us
Columns, Five Servings of Strength- a column by Michael Casten
An envelope covered in hearts and addressed to “Mom and Dad.” This envelope was kept secret for days, but ... Read more
April 17, 2018April 16, 2018
Adventures in the Big Apple (Part 1)
Columns, Embracing My Inner Alien - a column by Kevin Schaefer
Before I go any further with this column, let me go ahead and get this out of the way: ... Read more
April 16, 2018April 17, 2018
#AAN2018 – Olesoxime Stabilizes SMA Patients’ Long-term Movement Function, Extension Study Shows
News
Olesoxime (TRO19622) stabilizes the movement function of patients with Type 2 or Type 3 spinal muscular ... Read more
April 13, 2018April 12, 2018
For My Best Friend, My Brother
Columns, Life, One Cup at a Time - a Column by Alyssa Silva
On April 10, social media exploded with pictures and posts pinned with #NationalSiblingsDay. Using it, people shared sweet or ... Read more
April 13, 2018April 13, 2018
Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
News
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in ... Read more
April 12, 2018April 11, 2018
An Update on My Spinraza Journey
Columns, Refined By Fire - a Column by Ryan Berhar
I haven’t updated my Spinraza journey for a while because, well, nothing has happened. But we are getting down to ... Read more
April 12, 2018April 12, 2018
NIH Agency Pioneers Collaborative Research into Rare Diseases
News
A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed ... Read more
April 11, 2018April 10, 2018
‘Family Perspectives’: A Reflection on the Interviews
Columns, Five Servings of Strength- a column by Michael Casten
Editor’s note: Columnist Michael Casten caps his “Family Perspectives” series exploring how different people who surround Ella tackle the ... Read more
April 11, 2018April 11, 2018
Muscle Waste Product Creatinine Might Be Used as SBMA Biomarker, Study Reports
News
Blood levels of a waste product from muscle metabolism could be used to see how spinal and bulbar muscular atrophy ... Read more

April 20, 2018April 19, 2018

The Never-ending Quest to Gain Weight

Columns, Life, One Cup at a Time - a Column by Alyssa Silva

Although Mother Nature seems to have other plans lately, the days crossed off our calendars have us inching closer and closer to summer. Ah, yes, summertime, when the weather ... Read more

April 20, 2018April 20, 2018

#AAN2018 — Spinraza Improves Motor and Respiratory Function in SMA Children, Trials Show

News

Spinraza (nusinersen) improves motor and respiratory function in children with spinal muscular atrophy (SMA), according to results from four trials. Biogen will present the data at the American Academy of Neurology annual ... Read more

April 18, 2018

#AAN2018: SMA Treatment Candidate RG7916 Increases Amount of Key SMN Protein, Clinical Trials Show

News

Roche’s treatment candidate RG7916 increased the amount of full-length, functional SMN protein in spinal muscular atrophy (SMA) patients, according to soon-to-be-presented data from a Phase 2/3 clinical trial. The company will ... Read more

April 18, 2018April 17, 2018

A Message for Us

Columns, Five Servings of Strength- a column by Michael Casten

An envelope covered in hearts and addressed to “Mom and Dad.” This envelope was kept secret for days, but there were hints that it was coming. Ella worked on ... Read more

April 17, 2018April 16, 2018

Adventures in the Big Apple (Part 1)

Columns, Embracing My Inner Alien - a column by Kevin Schaefer

Before I go any further with this column, let me go ahead and get this out of the way: My parents are awesome, and I saw “Hamilton” on Broadway with ... Read more

April 16, 2018April 17, 2018

#AAN2018 – Olesoxime Stabilizes SMA Patients’ Long-term Movement Function, Extension Study Shows

News

Olesoxime (TRO19622) stabilizes the movement function of patients with Type 2 or Type 3 spinal muscular atrophy over the long term, an extension of a Phase 2 clinical trial ... Read more

April 13, 2018April 12, 2018

For My Best Friend, My Brother

Columns, Life, One Cup at a Time - a Column by Alyssa Silva

On April 10, social media exploded with pictures and posts pinned with #NationalSiblingsDay. Using it, people shared sweet or comical words about their siblings and what they mean to ... Read more

April 13, 2018April 13, 2018

Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy

News

A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, ... Read more

April 12, 2018April 11, 2018

An Update on My Spinraza Journey

Columns, Refined By Fire - a Column by Ryan Berhar

I haven’t updated my Spinraza journey for a while because, well, nothing has happened. But we are getting down to the wire. Something is about to happen — or not happen ... Read more

April 12, 2018April 12, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

News

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

April 11, 2018April 10, 2018

‘Family Perspectives’: A Reflection on the Interviews

Columns, Five Servings of Strength- a column by Michael Casten

Editor’s note: Columnist Michael Casten caps his “Family Perspectives” series exploring how different people who surround Ella tackle the diagnosis of her SMA. Read an introduction to the series here. Three generations ... Read more

April 11, 2018April 11, 2018

Muscle Waste Product Creatinine Might Be Used as SBMA Biomarker, Study Reports

News

Blood levels of a waste product from muscle metabolism could be used to see how spinal and bulbar muscular atrophy (SBMA) develops before symptoms appear, a Japanese study reports. The ... Read more

The Never-ending Quest to Gain Weight

#AAN2018 — Spinraza Improves Motor and Respiratory Function in SMA Children, Trials Show

#AAN2018: SMA Treatment Candidate RG7916 Increases Amount of Key SMN Protein, Clinical Trials Show

A Message for Us

Adventures in the Big Apple (Part 1)

#AAN2018 – Olesoxime Stabilizes SMA Patients’ Long-term Movement Function, Extension Study Shows

For My Best Friend, My Brother

Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy

An Update on My Spinraza Journey

NIH Agency Pioneers Collaborative Research into Rare Diseases

‘Family Perspectives’: A Reflection on the Interviews

Muscle Waste Product Creatinine Might Be Used as SBMA Biomarker, Study Reports

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