News

January 15, 2021January 15, 2021

Online for 2021, Team Cure SMA Race Series Planning Variety of Events

News

The COVID-19 pandemic has forced many activities to be online only, but in the case of this year’s Team Cure SMA endurance race series, the limitation means more people ... Read more

January 12, 2021January 13, 2021

Spinraza Trial Begins Treating Children Who Failed to Respond to Gene Therapy

News

The first patient has been dosed in a Phase 4 trial exploring the safety and potential benefits of Spinraza (nusinersen) in children with spinal muscular atrophy (SMA) who failed ... Read more

January 11, 2021January 11, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 8, 2021January 8, 2021

New Cure SMA Webinar Series to Focus on Health, Wellness

News

Cure SMA has developed a new three-part webinar series that delves into health and wellness topics pertinent to the spinal muscular atrophy (SMA) community. The three monthly installments of ... Read more

January 6, 2021January 6, 2021

Higher-dose Spinraza Trial Now Enrolling Part B After No Safety Issues Found

News

After finding no safety concerns in Part A of the DEVOTE trial, which is testing higher doses of Spinraza (nusinersen) among people of all ages with spinal muscular atrophy (SMA), ... Read more

January 4, 2021January 4, 2021

Informal SMA Caregivers Surveyed in Europe Report High Daily Burden

News

Non-professional caregivers who care for children with spinal muscular atrophy (SMA) carry a high daily burden that negatively affects certain aspects of their lives, according to a questionnaire study ... Read more

December 21, 2020December 21, 2020

Zolgensma Linked in 3 Cases to Serious But Treatable Blood Disorder

News

Some children with spinal muscular atrophy (SMA) may develop thrombotic microangiopathy (TMA) — a rare condition characterized by blood abnormalities — following treatment with Zolgensma, a study reports. A ... Read more

December 18, 2020December 18, 2020

Zolgensma Approved to Treat Young SMA Patients in Canada

News

Health Canada has approved Novartis‘ gene therapy Zolgensma (onasemnogene abeparvovec) to treat pediatric spinal muscular atrophy (SMA) patients who have three or fewer copies of the SMN2 gene, or with infantile-onset ... Read more

December 16, 2020December 16, 2020

Paramedian Injection Approach Found to Ease Spinraza Administration

News

Administering Spinraza (nusinersen) via a particular method of injection, called the paramedian approach, can shorten procedure times and reduce the occurrence of adverse events for people with spinal muscular ... Read more