News

August 11, 2020August 11, 2020

SMA Foundation’s ‘Little Toolbox’ Helped Pave Way to Therapies

News

[Editor’s Note: This is part of a series of articles into the discovery and development of Evrysdi, SMA’s newly approved disease-modifying therapy and its first oral and at-home one, ... Read more

August 11, 2020August 11, 2020

New Streaming Channel Showcases Rare Disease Films

News, syndicated

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the ... Read more

August 10, 2020August 10, 2020

‘Lucky Genetics’ and Hard Work: SMA’s Leap to 3 DMTs in 4 Years

News

August 7, 2020August 8, 2020

FDA Approves Risdiplam, as Evrysdi, 1st Oral Treatment for All SMA Types

News

The U.S. Food and Drug Administration (FDA) has approved Evrysdi — formerly known as risdiplam — as the first oral and at-home treatment for adults, children, and infants 2 months ... Read more

August 7, 2020August 7, 2020

Phase 2 Trial of Amifampridine in SMA Type 3 Patients Fully Enrolled

News

A clinical trial testing oral amifampridine phosphate in people with spinal muscular atrophy (SMA) type 3 who are able to walk is fully enrolled, Catalyst Pharmaceuticals announced in a press release. Marketed under ... Read more

August 5, 2020August 5, 2020

Cure SMA Offers Free Medical Alert Bracelet Kits to US Patients

News

Cure SMA has launched a new program to offer medical alert bracelets to spinal muscular atrophy (SMA) patients in the United States at no cost to the patient, according ... Read more

August 3, 2020August 3, 2020

New ASAH1 Mutations Linked to Rare Case of SMA and Farber Disease

News

A rare instance of a child with both spinal muscular atrophy (SMA) and Farber disease, associated with two new mutations in the ASAH1 gene, is detailed in a case report. Its ... Read more

July 31, 2020August 3, 2020

Spinraza-Zolgensma Combination Well-tolerated in Children with SMA Type 1, Study Shows

News

Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. The data, which ... Read more

July 29, 2020July 30, 2020

Plans Advance for Biomarkers Panel to Assess Drug-induced Skeletal Muscle Injury

News

A new panel of four safety biomarkers to assess acute drug-induced skeletal muscle injury in Phase 1 clinical trials has received a positive response from the U.S. Food and ... Read more

July 27, 2020July 27, 2020

Blocking Calpain Enzyme Found to Increase SMN Protein in Preclinical Models

News

In cell- and mouse-based models of spinal muscular atrophy (SMA), reduced levels of the SMN protein — a hallmark of the disease — altered both the calpain enzyme system ... Read more

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