Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) seeking approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 45 skipping. “The completion of our casimersen submission is an important milestone in our journey to advance treatments…
In the disabled community, many people discuss the negative implications of being called “inspiring.” Some have said it’s objectifying, reduces our humanness, or congratulates us just for being disabled. I agree with many of those statements; however, if I am helping a person work past their…
The COVID-19 pandemic has forced many to shelter at home, caused millions of layoffs, and tightened budgets. But for families affected by Duchenne muscular dystrophy (DMD), who front extra medical costs and must decide between working and potentially exposing their son to the virus, the pandemic…
The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a label update for Translarna (ataluren) to enable its use in Duchenne muscular dystrophy (DMD) patients who lost their ability to walk. The committee has specifically voted to remove…
“Life can only be understood backwards; but it must be lived forwards,” said philosopher Søren Kierkegaard. He came up with an existential masterpiece. In a dozen words, Kierkegaard captured what we all struggle with in finding meaning in our lives. I find it much easier to look backward to…
A Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy type 2i (LGMD2i), has dosed its first healthy volunteer, ML Bio Solutions announced. The company also announced that children and adults with a confirmed LGMD2i genetic diagnosis are invited to join a natural history…
It is undeniable that the coronavirus pandemic has affected many aspects of life. Along with establishing new norms for work, school, and social interaction, the pandemic has forced romantic relationships to undergo alterations. “When can I see you again?” my boyfriend, Will, asked for the…
A partnership between Muscular Dystrophy Canada (MDC) and Technology for Living (TFL) will seek to provide a cough assist device to any adult in British Columbia who has neuromuscular disease and needs one. Under the new partnership, adults who are registered with the…
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…
For a long time, I didn’t think journalism was a creative endeavor. I viewed the craft from an analytical perspective. After all, I was just recounting my firsthand experience publishing interviews and translating research. It wasn’t until recently — when I began reading the book “…
Get regular updates to your inbox.