July 1, 2020July 23, 2020
Into the Clearing: Emerging From the Fog of an SMA Diagnosis Sponsored Post
December 1, 2020November 24, 2020
Identity, SMA, and Thinking Outside the Box

In his essay for The New Yorker, “Who Am I?”, comedian Demetri Martin explores his identity both literally and figuratively. He opens by saying ... Read more
November 30, 2020November 23, 2020
My Period Reminds Me of the Inevitability of Disease Progression

Once every few weeks, I’m reminded of my own mortality. A lot of folks dread their period. It’s a definitively unpleasant time between the ... Read more
November 30, 2020November 30, 2020
Extensive Trial Monitoring Confirms Evrysdi Does Not Damage Vision

Evrysdi (risdiplam) at its approved and therapeutic dose does not lead to eye damage in children or adults with spinal muscular atrophy (SMA), data from extensive ... Read more
November 26, 2020November 26, 2020
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their ... Read more
November 25, 2020November 23, 2020
Being Thankful, No Matter What

It’s so much more than the turkey. Memories of childhood Thanksgiving gatherings with grandparents, aunts, uncles, and cousins take me to a happy place. ... Read more

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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 ... Read more

Identity, SMA, and Thinking Outside the Box

My Period Reminds Me of the Inevitability of Disease Progression

Extensive Trial Monitoring Confirms Evrysdi Does Not Damage Vision

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Being Thankful, No Matter What

Extensive Trial Monitoring Confirms Evrysdi Does Not Damage Vision

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Being Thankful, No Matter What

Ella Has Fun While Sheltering at Home

Cure SMA Offering Free Masks, Other COVID-19 Equipment to Patients

To the People Who Got Me Through This Year

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Olesoxime Failed to Benefit SMA Type 2 and 3 Patients, Final Trial Data Show

A Sigh of Gratitude After November’s Golden Yawn

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Ella Has Made Big Gains Since Diagnosis Day

SMA Mutations Impact Development, Protein Production in Prenatal Mice

It’s Been an SMA Kind of Day

Gene Therapy Given Directly to Spinal Canal Might Be Safer With ‘Silencing’ Step

My Life Uniquely Allows Me to Be Naturally Empathetic

Life With SMA Can Feel Like an Anomaly

Spinraza Shown to Preserve Respiratory Muscle Strength in SMA Type 2 Patients

Spinraza Shown to Preserve Respiratory Muscle Strength in SMA Type 2 Patients

Remembering That November, 23 Years Ago

Ella’s Wheelchairs Became Simpler Over Time

Lung Health Declines in Step With Motor Loss in Types 2 and 3, Study Finds

When It Rains, It Pours

Spinraza’s Benefits Mild, Transient in Infant With Severe Type 0 SMA

Molecule Similar to Evrysdi But Possibly Safe at Higher Doses Identified

Some Trails Are Buddy Trails

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

The Casten Family Prepares for a Different Kind of Holiday Season

Muscle MRI Captures Spinraza’s Effects on Tissue Fibers in Type 3 Brothers

I’ve Learned That My Body Requires Balance

‘Livin’ It Up on Top’: To the Worlds We Dream About

Screening for Carriers of SMA May Be Cost-free Option for Couples in Australia

People With Disabilities Need the Freedom to Explore Their Interests

NICE Reviews UK Spinraza Reimbursement for SMA Type 3 Patients Unable to Walk

When the Scary Stuff Became Real

Muscle-directed Therapy SRK-015 Improves Motor Function in SMA Types 2, 3, Data Show