March 5, 2021March 5, 2021
Challenges of Life as Teen, Young Adult With SMA Highlighted in Survey

Being an adolescent or young adult with spinal muscular atrophy (SMA) presents unique challenges, according to a study based on viewpoints expressed by patients ages ... Read more
March 4, 2021March 2, 2021
Hot Air Balloons Adrift: Appreciating My Pulmonary Function With SMA

I gotta go climb some mountains, and I gotta do it now. That was the thought I focused on as I emerged from the pulmonary ... Read more
March 3, 2021March 1, 2021
Mama, It’s Amazing What SMA Patients Can Do: A Reminder of Our Adaptability

My neurologist presses his stethoscope to my chest. He’s performing a routine checkup; it’s a mere formality, really, so I can be sent home with ... Read more
March 3, 2021March 3, 2021
CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment

Evrysdi (risdiplam) has been recommended for approval in the European Union (EU) to treat spinal muscular atrophy (SMA) patients, ages 2 months and older, ... Read more
March 1, 2021February 25, 2021
My SMA Friends Remind Me That It’s OK to Rest

You already know that for the longest time, I didn’t want friends with SMA. I was young and desperate to be seen as “cool” (whatever ... Read more

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

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, researchers suggest. The study, “Seamless genetic conversion of SMN2 to SMN1 ... Read more

Challenges of Life as Teen, Young Adult With SMA Highlighted in Survey

Hot Air Balloons Adrift: Appreciating My Pulmonary Function With SMA

Mama, It’s Amazing What SMA Patients Can Do: A Reminder of Our Adaptability

CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment

My SMA Friends Remind Me That It’s OK to Rest

Mama, It’s Amazing What SMA Patients Can Do: A Reminder of Our Adaptability

CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment

My SMA Friends Remind Me That It’s OK to Rest

Evrysdi Seen to Improve Survival, Motor Development of Infants with SMA Type 1

You Can Ask Me About My Disability Any Day

My Unsolved Symptoms Add Flair to My Rare

Subscribe to our mailing list

Rare Diseases and Flying Under the Radar to Pleasantly Surprise People

Being Part of the ‘Rare’ Family Brings Unforeseen Blessings

Cure SMA Offers Free Virtual Therapy Sessions

This Rare Disease Day, I’m Grateful for My Friends

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

How a Head Cold Saved My Life (Figuratively)

Study Finds Varying Perspectives From Parents on Spinraza Therapy for Children

Searching for the Wisdom to See Challenges in New Ways

Learning to Cherish My Fortunes in the Year of the Ox

Learning to Cherish My Fortunes in the Year of the Ox

New SMN-boosting Molecule Shows Promise as Add-on Therapy

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

‘Don’t Tell Me What I Can’t Do’: How ‘Lost’ Navigates Disability

Rare Case of SMA Linked With Myoclonic Epilepsy Detailed in Report

10 Years Later, I’m Looking Back on My Last Hospitalization

My Hopes and Fears Resurface When Others Start SMA Treatment

Assistive Devices Should Be Standard in Managing SMA Type 1

Thriving in the Winter Pines: Mother Nature, Will You Be My Valentine?

No Backup Is Needed With Angel Intervention

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Quantitative MRI Is Sensitive Measure of Muscle Decline

How ‘Disability Visibility’ Illustrates Diversity and Equity

For the Love of Frozen Breakfast Food

Spinraza Improves Motor Function in Case of Teenager with Late-onset SMA

A ‘Miracle’: Spinraza Gives Boy, Now 5, a Chance to Grow Up

A Groundhog With SMA and a Writing Machine Considers Her Shadow

How CDPR’s ‘Cyberpunk 2077’ Relates to Disability

Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy

Challenges of Life as Teen, Young Adult With SMA Highlighted in Survey

Hot Air Balloons Adrift: Appreciating My Pulmonary Function With SMA

Mama, It’s Amazing What SMA Patients Can Do: A Reminder of Our Adaptability

CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment

My SMA Friends Remind Me That It’s OK to Rest

Mama, It’s Amazing What SMA Patients Can Do: A Reminder of Our Adaptability

CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment

My SMA Friends Remind Me That It’s OK to Rest

Evrysdi Seen to Improve Survival, Motor Development of Infants with SMA Type 1

You Can Ask Me About My Disability Any Day

My Unsolved Symptoms Add Flair to My Rare

Subscribe to our mailing list

Rare Diseases and Flying Under the Radar to Pleasantly Surprise People

Being Part of the ‘Rare’ Family Brings Unforeseen Blessings

Cure SMA Offers Free Virtual Therapy Sessions

This Rare Disease Day, I’m Grateful for My Friends

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

How a Head Cold Saved My Life (Figuratively)

Study Finds Varying Perspectives From Parents on Spinraza Therapy for Children

A Rare Disease Traveler Finds Her Village

Searching for the Wisdom to See Challenges in New Ways

Learning to Cherish My Fortunes in the Year of the Ox

Learning to Cherish My Fortunes in the Year of the Ox

New SMN-boosting Molecule Shows Promise as Add-on Therapy

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

‘Don’t Tell Me What I Can’t Do’: How ‘Lost’ Navigates Disability

Rare Case of SMA Linked With Myoclonic Epilepsy Detailed in Report

10 Years Later, I’m Looking Back on My Last Hospitalization

My Hopes and Fears Resurface When Others Start SMA Treatment

Assistive Devices Should Be Standard in Managing SMA Type 1

Thriving in the Winter Pines: Mother Nature, Will You Be My Valentine?

No Backup Is Needed With Angel Intervention

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Quantitative MRI Is Sensitive Measure of Muscle Decline

How ‘Disability Visibility’ Illustrates Diversity and Equity

For the Love of Frozen Breakfast Food

Spinraza Improves Motor Function in Case of Teenager with Late-onset SMA

A ‘Miracle’: Spinraza Gives Boy, Now 5, a Chance to Grow Up

A Groundhog With SMA and a Writing Machine Considers Her Shadow

How CDPR’s ‘Cyberpunk 2077’ Relates to Disability