SMA News Today

October 19, 2021October 19, 2021

Global Genes, Diversity Coalition Team Up to Advance Health Equity

SMA News Today

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, ... Read more

October 18, 2021October 18, 2021

Anxiety, Depression a Problem for School-aged Patients in China

SMA News Today

School-age children and adolescents with spinal muscular atrophy (SMA) in China experience a high rate of anxiety and depression, a questionnaire-based study suggests. “Professional psychological care included in ... Read more

October 15, 2021October 15, 2021

Novartis Wraps Zolgensma Price, Availability Negotiations in Canada

SMA News Today

Novartis has completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding pricing and availability of Zolgensma (onasemnogene abeparvovec), its approved gene therapy for spinal muscular atrophy (SMA), for pediatric patients. ... Read more

October 13, 2021October 13, 2021

Newborn Screening Pilot for SMA in Belgium Grows Into Official Program

SMA News Today

A pilot newborn screening (NBS) program for spinal muscular atrophy (SMA) that started in Liège province has transitioned into an official, government-run program spanning Southern Belgium. Its architects described their experience, ... Read more

October 12, 2021October 12, 2021

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

COVID-19 News, SMA News Today

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC ... Read more

October 11, 2021October 11, 2021

SMA Doesn’t Stop Teen From Pursuing Academic Dreams at MIT

SMA News Today

Ben Lou battles the weakness in his body that comes with having spinal muscular atrophy (SMA) type 2, but his mind is stronger than most: The 18-year-old was accepted to three ... Read more

October 8, 2021October 8, 2021

Cure SMA Advocates for Better Wheelchair Storage for Air Travel

SMA News Today

In a letter to a U.S. Department of Transportation Advisory Committee, Cure SMA called for better wheelchair handling and storage for people with spinal muscular atrophy (SMA) who travel by air. ... Read more

October 6, 2021October 6, 2021

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

SMA News Today

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and ... Read more

October 6, 2021October 6, 2021

Canadian Agency Favors Zolgensma Only for Babies Up to 6 Months Old

SMA News Today

The Canadian Agency for Drugs and Technologies in Health (CADTH) will not recommend reimbursement for Zolgensma in children with spinal muscular atrophy (SMA) who are more than 6 months old, citing ... Read more

October 4, 2021October 4, 2021

Phase 3 Apitegromab Trial Planned for Non-ambulatory Types 2, 3

SMA News Today

Scholar Rock plans to soon initiate a Phase 3 clinical trial of apitegromab, its investigational muscle-directed treatment for spinal muscular atrophy (SMA), in type 2 and 3 patients who are unable ... Read more

October 1, 2021October 1, 2021

Neurofilament Blood Levels Likely to Predict Treatment Response in SMA

SMA News Today

In infants with spinal muscular atrophy (SMA) type 1, lower blood levels of phosphorylated neurofilament heavy chain (pNF-H) — a marker of nerve cell damage — before starting treatment with Spinraza ... Read more

September 29, 2021September 29, 2021

New Quality of Life Survey Aims to Help Inform SMA Community Needs

SMA News Today

BioNews Insights is launching a new survey, in collaboration with SMA News Today, with the goal of understanding how different aspects of this disease affect an individual’s quality of life. ... Read more

September 28, 2021September 28, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

SMA News Today

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 27, 2021September 27, 2021

Pre-symptomatic Infants Retain Swallowing Ability in Evrysdi Trial

SMA News Today

After being treated with Evrysdi (risdiplam) for at least a year, pre-symptomatic infants with spinal muscular atrophy (SMA) have retained the ability to swallow, and most have been able to ... Read more

September 24, 2021September 24, 2021

Spinraza RESPOND Trial Enrolling Children Not Helped by Zolgensma

SMA News Today

Biogen is continuing to actively recruit patients for its Phase 4 RESPOND trial, which is evaluating the benefits of Spinraza (nusinersen) in infants and children with spinal muscular atrophy (SMA) who responded poorly to ... Read more