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

October 19, 2017October 19, 2017
#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
News
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act ... Read more
October 18, 2017
Tools to Help SMA Individuals: The JACO Robotic Arm
Social Clips
While there are many different types of adaptive equipment that can help SMA individuals and people with other forms ... Read more
October 18, 2017October 17, 2017
The Middle Child
Columns, Five Servings of Strength- a column by Michael Casten
The middle child holds a special place in everybody’s heart — they’re the peacekeeper, the negotiator, the one most ... Read more
October 18, 2017
Filmmakers’ Documentary on Pivotal Time in SMA Treatments Nears Fundraising Deadline
News
Three filmmakers are raising funds for a feature-length documentary about how science and the rare disease community’s needs came ... Read more
October 17, 2017October 18, 2017
Potential SMA Therapy, RG7916, for Types 2 and 3 Advancing in Clinical Trial in Europe
News
A Phase 2 clinical trial evaluating the efficacy and safety of RG7916 in children and adults with type ... Read more
October 17, 2017October 16, 2017
Never Tell Me the Odds
Columns, Embracing My Inner Alien - a column by Kevin Schaefer
In a few days I’ll be 24, and every year my birthday is accompanied with at least some sentimentality. ... Read more
October 16, 2017October 16, 2017
4 Things to Help with Depression
Columns, The Wolf Finally Frees Itself - a column by Brianna Albers
It’s easy for me to hide behind a computer screen and tell you that it gets better. To believe ... Read more
October 13, 2017October 12, 2017
The Perfect Bite-Sized Amount
Columns, Life, One Cup at a Time - a Column by Alyssa Silva
Until I was 15, I managed to stay reasonably healthy. But for the first time in almost a decade ... Read more
October 12, 2017
Q&A With Kevan Chandler: Adventurer, SMA Author and Nonprofit Founder
Social Clips
Kevan Chandler always dreamed of going to Europe, but knew that he’d have to develop a creative solution in ... Read more
October 12, 2017October 13, 2017
Doctoral Student Invents Tools to Help Herself and Others with Disabilities
News
Kavita Krishnaswamy is so determined to gain independence for herself and others living with physical disabilities that she spends ... Read more
October 11, 2017October 10, 2017
Always Work to Be Done
Columns, Five Servings of Strength- a column by Michael Casten
Ella’s a hard-working 7-year-old. She rises early in the morning for school. While at school, she does what all ... Read more
October 10, 2017October 9, 2017
Anxieties About Dating, the Friend-Zone, and Learning to Embrace Myself for Who I Am
Columns, Embracing My Inner Alien - a column by Kevin Schaefer
I’ve been writing this column since April, and while I haven’t shied away from addressing personal topics like adapting ... Read more

October 19, 2017October 19, 2017

#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies ... Read more

October 18, 2017October 17, 2017

The Middle Child

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

The middle child holds a special place in everybody’s heart — they’re the peacekeeper, the negotiator, the one most likely to feel overlooked. Henry fits the bill to a ... Read more

October 18, 2017

Filmmakers’ Documentary on Pivotal Time in SMA Treatments Nears Fundraising Deadline

News

Three filmmakers are raising funds for a feature-length documentary about how science and the rare disease community’s needs came together to make Biogen’s Spinraza the first approved treatment for spinal muscular ... Read more

October 17, 2017October 18, 2017

Potential SMA Therapy, RG7916, for Types 2 and 3 Advancing in Clinical Trial in Europe

News

A Phase 2 clinical trial evaluating the efficacy and safety of RG7916 in children and adults with type 2 or 3 spinal muscular atrophy has advanced into a second ... Read more

October 17, 2017October 16, 2017

Never Tell Me the Odds

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

In a few days I’ll be 24, and every year my birthday is accompanied with at least some sentimentality. I hate being sappy, and anytime I reach a milestone ... Read more

October 16, 2017October 16, 2017

4 Things to Help with Depression

Columns, The Wolf Finally Frees Itself - a column by Brianna Albers

It’s easy for me to hide behind a computer screen and tell you that it gets better. To believe that it gets better – yet still wake up every morning ... Read more

October 13, 2017October 12, 2017

The Perfect Bite-Sized Amount

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

Until I was 15, I managed to stay reasonably healthy. But for the first time in almost a decade that year, I was hospitalized. The winter months can be ... Read more

October 12, 2017October 13, 2017

Doctoral Student Invents Tools to Help Herself and Others with Disabilities

News

Kavita Krishnaswamy is so determined to gain independence for herself and others living with physical disabilities that she spends up to 20 hours a day designing devices. “After I ... Read more

October 11, 2017October 10, 2017

Always Work to Be Done

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

Ella’s a hard-working 7-year-old. She rises early in the morning for school. While at school, she does what all of the other kids do and more. She has therapies ... Read more

October 10, 2017October 9, 2017

Anxieties About Dating, the Friend-Zone, and Learning to Embrace Myself for Who I Am

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

I’ve been writing this column since April, and while I haven’t shied away from addressing personal topics like adapting to my weakening body and at times feeling burdensome, dating ... Read more

#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

Tools to Help SMA Individuals: The JACO Robotic Arm

The Middle Child

Filmmakers’ Documentary on Pivotal Time in SMA Treatments Nears Fundraising Deadline

Potential SMA Therapy, RG7916, for Types 2 and 3 Advancing in Clinical Trial in Europe

Never Tell Me the Odds

4 Things to Help with Depression

The Perfect Bite-Sized Amount

Q&A With Kevan Chandler: Adventurer, SMA Author and Nonprofit Founder

Doctoral Student Invents Tools to Help Herself and Others with Disabilities

Always Work to Be Done

Anxieties About Dating, the Friend-Zone, and Learning to Embrace Myself for Who I Am