Author name: Zach Galati

Rare Disease Month 2025: Driving Progress Through Research

February marks Rare Disease Month, a time to amplify the voices of the 300 million people worldwide living with a rare disease—including the individuals in the U.S. impacted by spinal muscular atrophy (SMA). Throughout this month, and especially during Rare Disease Week (starting February 23rd), we’re committed to advancing research, raising awareness, and advocating for […]

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All 2025 Walk-n-Roll Events Are Open

Cure SMA warmly invites you to join us at one of our 2025 Walk-n-Roll events—where community meets impact! This year, for the first time, you can register and start fundraising for both spring and fall events right now. Walk-n-Roll isn’t just a fundraiser; it’s a celebration of progress, hope, and togetherness. It’s a chance to

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Biohaven Provides Update on Taldefgrobep Alfa Development Program for Spinal Muscular Atrophy

In the RESILIENT SMA study, Biohaven announced today that taldefgrobep alpha showed clinically meaningful improvements in motor function at all time points on the Motor Function Measurement-32 scale (MFM-32), but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care (SOC) group. Biohaven plans to

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Big Week in Washington for Cure SMA-Supported Legislation

This week, bills to address everyday challenges faced by individuals with spinal muscular atrophy (SMA) took important steps forward in the U.S. Congress. A key aviation bill that includes a section that will make air travel safer and more accessible for passengers with disabilities, including SMA, was approved by the U.S. House of Representatives by

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Cure SMA’s 2022 State of SMA Report

Cure SMA is pleased to announce the launch of the second annual State of SMA report. The purpose of this report is to share highlights from Cure SMA’s three databases: a patient-reported database with data from over 9,700 affected individuals worldwide that also incorporates longitudinal data from our annual community update survey; the SMA clinical

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Individuals with SMA Describe New Travel Hiccups and Wheelchair Damage in Their Air Travels to Cure SMA National Conference

A record-number of individuals with spinal muscular atrophy (SMA), a neuromuscular disease, travelled to Orlando to attend Cure SMA’s national conference from June 29 to July 2. Several adults with SMA and families of children with SMA reported damage to their wheelchairs and other air travel hiccups that impacted their conference experience. These latest travel

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