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Developing Effective, Safe, and Accessible New Drugs for Rare Disorders in...

An article in the January issue of Therapeutic Innovation and Regulatory Science (formerly the Drug Information Journal) offers continuing insights into how the pharmaceutical and biotechnology...

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Orphan Drug Act at 30: Will Success Become Too Expensive?

Reprinted with permission from FDA Matters:  By Steven Grossman The Orphan Drug Act (ODA) turned 30 this month, demonstrating that good laws really can have an enduring impact.  Amidst the...

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Raise Your Hand to Support Rare Disease Research

The fourth annual Raise Your Hand campaign launched this week, and it’s already creating a social media buzz.   This campaign, sponsored by Lundbeck, raises awareness and support for rare disease...

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Stiff Person Syndrome: A Fight to the Finish Line

My name is Debbie Richardson, and I was diagnosed in 1994 with Stiff Person Syndrome (SPS), following four years of progressive symptoms.  SPS is a rare autoimmune neurological disorder with an...

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Working Through the Math of the NIH Budget for Rare Disease Research: Charts...

Take Action Now to Support Rare Disease Research Now that the election is well behind us, talks have resumed in Washington, DC about the Federal budget.  To recap — the only major piece of the larger...

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What Does the 2013 Rare Disease Day Slogan Mean to You?

This year’s theme for Rare Disease Day is “Rare Disorders Without Borders.” It shows that rare diseases are a global issue, and if we work together in solidarity, we can create a better world for...

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Living With A Rare Disease

We know the math:  In the U.S., a disease is rare if it affects fewer than 200,000 people. There are over 7,000 rare diseases. Taken together, these rare diseases affect between 25 and 30 million...

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Today is Rare Disease Day. Will You Show Your Support?

Today is the day when people around the world will be posting stories, photos and videos about what it means to live with a rare disease.  From Kalamazoo to Kiev, the last day of February is observed...

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New Guidelines on Testing Kids’ DNA–the Cliff’s Notes Version

Exomes are big news. Sequencing of the protein-encoding part of the genome is increasingly solving medical mysteries in children. It began with Nicholas Volker and his recovery from a devastating...

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Francis Collins on developing drugs for rare disorders.

If you’ve never seen or heard NIH Director Francis Collins talking about drug development — and specifically the development of new drugs for rare disorders — just click here to see his TED...

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President Obama Announces “Brain Initiative”

Image Credit: Reuters/Jason Reed I represented NORD at the White House this morning at an event where President Obama announced the launch of a “Brain Initiative” to help researchers find new ways to...

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Borders Beyond Borders

Rare Disease Day organizers from around the world made “Rare Disorders Without Borders” the slogan for 2013. I see in it a potent reminder that rare disorders exist everywhere in the world, and I see...

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Making Sense of Antisense Therapy

What is antisense therapy? Most human diseases are caused by production of abnormal proteins or malfunctioning proteins.   Antisense therapy involves inhibiting production of these proteins.  When a...

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Heroes at the Boston Marathon

I originally started writing this blog days before Boston Marathon 2013, and was planning to post it after returning home.  I’m glad that I hadn’t posted it beforehand, because the terrible events at...

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The Borders Within

The slogan, “Rare Disorders without Borders” conjures geographical borders—the borders between cities, states, nations, continents. Ideally, where a person is on a map should not factor into the...

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Rare Disease Researchers – They Really Do Care!

In my role as NORD’s research grant program administrator, I’m in the unique position to frequently speak with many researchers.  Whether they are applying for one of our scientific seed grants...

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Entertainer for NORD’s Gala Shows Support for the Rare Disease Community

Siobhan Magnus’ talent as a vocalist provided her a national platform, and she says she is delighted to use those talents in ways she would never have imagined. Siobhan has been singing since she was a...

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RareConnect launches 40th online community!

Over the last 3 years our team has worked hard to expand the RareConnect project and we are thrilled to announce we now have over 40 active global, disease specific communities with the launch of the...

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The best part of my job.

I love my job, but the best part of my job is connecting rare disease patients. I was hired in June of 2011 to manage RareConnect- NORD’s global, online communities project with EURORDIS. About eight...

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High-School Students Raise Awareness

Today, members of the NORD staff had the opportunity to meet a group of high-school advocates who are already making a difference to help raise awareness for rare diseases. The students are all members...

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