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December 4, 2013

TOPIC: Medical, Research

A CRISPR Approach to Genome Editing

Posted at December 12, 2013 04:30 pm by Marsha Lanes

Genome editing is a process that involves cutting out mutations in DNA and replacing them with new genetic material.  This is an exciting idea because if it can be refined to a level that is safe and effective, the prospect of eradicating genetic diseases is no longer science fiction.  Read More

October 23, 2013

TOPIC: Advocacy, Featured News, Medical, Patients & Members

30 Years After the Orphan Drug Act: It Still Takes Too Long for People with Rare Diseases to Get an Accurate Diagnosis

Posted at October 10, 2013 02:44 pm by Mary Dunkle

 

NORD recently posted the following question on its Facebook page: How long did it take you or a loved one to get an accurate diagnosis?

We were stunned at the response. Within the first few hours, we got 200 replies and 20% of those who responded had waited 10 years or longer to get an accurate… Read More

August 1, 2013

TOPIC: Medical, Patients & Members, Research

Informed Consent in Biobank Research

Posted at August 8, 2013 06:33 pm by Marsha Lanes

Genetic biobanks, storage facilities for DNA, genetic data and tissue samples, are becoming more prevalent in the rare disease community.   The information and samples can be made available to approved researchers for clinical studies, but unlike clinical trials, there is usually no personal benefit or follow-up provided to participants.  (more…)Read More