Activity

Treatment with CAP-1002 improves respiratory, cardiac and muscle function in boys and young men with advanced DMD, final data from the HOPE-2 trial show. Read more here.

What do you think of these findings?

Hey everyone! Hope you all had a safe and relaxing weekend. For those here in the U.S., I wish you all a good Memorial Day.

So, as states and other places around the world begin to reopen, I wanted to get your thoughts. I don’t want to start anything political here. Rather, I’m just curious if anyone here feels comfortable going out som…[Read more]

Some 1,500 delegates from 57 countries joined the virtual European Conference on Rare Diseases this month with a focus on unity and standardized policies. Read more about the conference here.

What are other important issues you think the global rare disease community should address?

Neuromuscular specialists in the U.S. provided recommendations to help Duchenne and Becker MD patients deal with the challenges of the COVID-19 pandemic. Click here to read more.

Did you find these recommendations useful?

The NIH-led research network hopes for 5,000 responses to its online survey of how people with rare diseases in the US are being affected by the pandemic. Click here to learn more about the survey and how you can participate in it.

How has the global pandemic affected you? What do you think the survey will reveal about the pandemic’s…[Read more]

So I’m sure you’re all pretty tired of hearing about COVID. Paying attention to the news right now is exhausting.

However, I did want to share this column about COVID-19 and post-traumatic growth. The author, Brianna Albers, is a writer for one of our sibling sites. She’s been writing about mental health coping mechanisms, speci…[Read more]

Click here to read NORD’s call to action for rare disease patients on financial and legislative issues in the wake of the COVID-19 pandemic.

Which of these issues is most important to you and why?

Hey everyone! Hope you all had a safe and relaxing weekend.

Today, I wanted to start a conversation about setting personal goals. Obviously, it’s an unusual time to even think about this subject. Between the COVID-19 pandemic and just trying to stay healthy, most of us aren’t focused on the future.

Still, I actually think it’s a great…[Read more]

Click here to read about $1 million in seed funding to support work by Myosana Therapeutics on a potential non-viral and full-length dystrophin gene therapy for DMD patients.

What do you think of this news?

Since the COVID-19 pandemic is having a major impact on our healthcare services, we have to approach medical appointments differently than before. TeleMed and other virtual services could become the new norm. Still, some appointments and procedures need to be in-person. It raises questions about how we can best approach appointments with our…[Read more]

Treatment for six months with Increlex hormone therapy improved growth but not muscle function in boys with DMD, an early study found. Learn more here.

What do you think of this study and its findings?