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Blog Post | News

NALC to Deliver the Cure for MDA on #GivingTuesday

Since 1952, the National Association of Letter Carriers, the nationwide union of US postal delivery workers, has supported MDA as its charity of choice β€” and raised more than $100…

Tags: Fundraising, MDA Care Centers, Summer Camp


Blog Post | Personal Stories

Fire Fighter, Father, Friend: JR Ortiz Bolsters MDA, From Boston to New York City

Santos β€œJR” Ortiz hadn’t always dreamed of becoming a fire fighter. He didn’t play on fire trucks as a kid, or even know much about fire departments. But when he…

Tags: Ambassadors, Summer Camp, Team Momentum


Blog Post | News, Science + Research

Roche Genentech Announces Positive Results from Pivotal Study of Risdiplam in SMA Types 2 and 3

On Nov. 10, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 SUNFISH clinical trial assessing risdiplam in patients with…

Tags: Clinical Trials, Drug Development, Research, Research Advances


Blog Post | News, Science + Research

Solid Biosciences Releases Letter to DMD Community Announcing Hold on IGNITE DMD Trial Due to a Serious Adverse Event

Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding the decision by the US Food and Drug Administration (FDA) to place the company’s IGNITE DMD…

Tags: Clinical Trials, Research


Blog Post | Get Involved, News

What’s #GivingTuesday All About?

#GivingTuesday, annually held immediately after the Black Friday and Cyber Monday holiday shopping days following Thanksgiving, falls late this year on Dec. 3. But here at MDA, we’re already planning…

Tags: Clinical Trials, Drug Development, Fundraising


Blog Post | News, Science + Research

Roche Genentech Releases Letter to DMD Community Announcing Discontinuation of Clinical Trials Program for DMD

Today, Roche Genentech released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full letter follows. Letter to the…

Tags: Clinical Trials, Research


Blog Post | News, Science + Research

MDA Care Centers Combine Expert Care with Research, Clinical Trials, and Advanced Technology

At more than 150 of the leading healthcare institutions across the US, MDA Care Centers offer top clinical care to individuals living with muscular dystrophy, ALS, and other neuromuscular diseases.…

Tags: Clinical Trials, Grants, MDA Care Centers, Research


Blog Post | News, Science + Research

AveXis Releases Letter to SMA Community: FDA Places Partial Hold on Clinical Trials for Intrathecally Delivered AVXS-101

On Oct. 30, AveXis released a letter to the spinal muscular atrophy (SMA) community regarding a decision by the US Food and Drug Administration (FDA) to place a partial hold…

Tags: Clinical Trials, Research


Blog Post | News, Science + Research

World Muscle Society Roundup: Clinical Trial Results of Treatments in Development for DMD, XLMTM, SMA, and Pompe Disease

Several biotech and pharma companies presented interim results at the 24th Annual Congress of the World Muscle Society (WMS) held earlier this month in Copenhagen, Denmark. This roundup covers a…

Tags: Clinical Trials, Research


Blog Post | News, Science + Research

Reata Pharmaceuticals Announces Positive Results from Pivotal Study of Omaveloxolone in FA

Texas-based Reata Pharmaceuticals announced positive results of its pivotal phase 2 clinical trial (MOXIe) of omaveloxolone in patients with Friedreich’s ataxia (FA). Patients treated with omaveloxolone showed a statistically significant…

Tags: Clinical Trials, Drug Development, Research