3-D printing is an exciting technology that has shown great promise in the treatment of several rare disorders. Bioprinting is a form of 3-D printing in which cells are printed or deposited on a surface layer by layer in order to produce a full organ. Lab-built organs have been shown to function well in patients, and engineered airways, bladders, blood vessels and urine tubes have been successfully implanted. An airway splint printed from biomaterials and without cells was used to save the life of an infant with tracheobronchomalacia, a rare condition where the tracheal (windpipe) cartilage is soft. The FDA approved the use of the device under the emergency-use exemption and the case report was described in the New England Journal of Medicine. read more >
There have long been clinicians and researchers with specific interests in rare and “orphan” disorders.
One of the first such clinicians who some readers may know of was Sir Archibald Garrod. Garrod was a British physician who first characterized alkaptonuria or “black urine disease” in 1902. What is more, he identified this disorder as being an inborn error of metabolism (i.e., a genetic disorder). Garrod’s early studies in this area led to the development of a whole new branch of medicine. read more >
NORD recently hosted a Capitol Hill briefing on the topic “Rare Diseases in a Changing Healthcare Landscape”. Keeping our elected officials informed and aware is a critically important, and this briefing was very well received. read more >
I woke up this morning in a great mood. Something wonderful happened last night. Let’s see, what was it? Oh yeah, that Jeopardy question. Ugh. Then there was the realization that the moment was over, for good. Done and gone. So fleeting fame can be. read more >
NORD has submitted the following Letter to the Editor of the Journal of the American Medical Association (JAMA):
The recent study evaluating the evidence supporting FDA approval of novel therapeutic agents has generated debate about FDA exercise of flexibility and judgment in approving new drugs. FDA Commissioner Margaret Hamburg, MD, wrote a blog explaining why FDA supports a flexible approach to drug development.  read more >
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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