News

March 11, 2021March 11, 2021

MDA Engage Offers Free Educational Events

News

Through a program called MDA Engage, the Muscular Dystrophy Association (MDA) is offering virtual educational events for people affected by muscular dystrophy and other neuromuscular diseases. The events are aimed ... Read more

March 9, 2021March 12, 2021

#MDA2021 – MDA Virtual Conference to Feature Latest in Clinical Care, Research

MDA 2021, News

Next week’s 2021 MDA Virtual Clinical and Scientific Conference is expected to attract more than 1,200 clinicians and researchers from across the U.S. with expertise in neuromuscular conditions. Hosted by the Muscular ... Read more

March 5, 2021March 9, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

News, syndicated

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 4, 2021March 4, 2021

Vamorolone Trial for DMD Finishes Pivotal First Part, Results Awaited

News

The final patient has finished the first, six-month portion of the pivotal VISION-DMD Phase 2b clinical trial, assessing the anti-inflammatory steroid therapy vamorolone in boys with Duchenne muscular dystrophy (DMD). ... Read more

March 2, 2021March 2, 2021

Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i

News

The first patient has been dosed in a Phase 2 trial testing BBP-418, a potential first oral disease-modifying therapy for limb-girdle muscular dystrophy type 2i (LGMD2i). BBP-418 is a small molecule that ... Read more

February 26, 2021February 26, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

News, syndicated

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more

February 25, 2021February 25, 2021

Exon Skipping Dominates DMD Treatment, But What’s on Horizon?

News

With the recent approval of Amondys 45 (casimersen) by Sarepta Therapeutics, there are now four exon-skipping therapies available in the U.S. for eligible patients with Duchenne muscular dystrophy (DMD). The others are Exondys ... Read more

February 25, 2021February 26, 2021

FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation

News

The U.S. Food and Drug Administration (FDA) has conditionally approved Amondys 45 — formerly known as casimersen — by Sarepta Therapeutics as the first treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 45 ... Read more

February 23, 2021February 23, 2021

Givinostat Continues to Delay DMD Progression in Long-term Study, New Data Show

News

Treatment with givinostat alongside corticosteroids continues to delay disease progression — and maintain patients’ ability to walk — in boys with Duchenne muscular dystrophy (DMD), new data from a long-term ... Read more

February 22, 2021February 22, 2021

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

News, syndicated

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a ... Read more