A Collection of Rare Disease Posts

Rare Disease Day is held to raise much needed awareness to rare or orphan diseases. In order to qualify as a rare disease, its incidence rate must affect less than 200,000 people as established by the Congress of the United States with the Orphan Drug Act of 1983.1  In fact, the legislation, in addition to creating the definition, was also instrumental in changing the entire discussion and economy around orphan drug research in the US.  For example, during the ten years prior to its implementation, less than ten treatments were approved. However, since1983, the Office of Orphan Products Development (OOPD), which incentivizes research on what were previously untenable developmental costs, has been integral to bringing more than 400 new treatments to market. 1

There are believed to be upwards as many as 7,000 rare diseases according to the NIH.  Within their numerous centers, one, the National Center for Advancing Translational Sciences (NCATS), studies commonalities found among populations and diseases through collaborative studies.1 From that, the following programs for rare diseases have been made available:

Of course, with increased and vigorous reporting, collaborations, computer models, and even communications, the designations and prevalence of rare, and in fact, all diseases may change over time.

Here are a collection of Rare Disease Features from our archives:

"Rare Disease Journal Inaugural Edition - BBS"

A look at Bardet Biedl Syndrome and rare disease research products.

 

Rare Diseases Journal II, Sanfilippo Syndrome

A PepTalk on a devastating pediatric lysosomal storage diseases.

 

Rare Diseases Journal III - Von Hippel-Lindau Disease

VHL is s typified by abnormal growth of both benign and cancerous tumors which generally begin to appear in young adulthood.

 

Scorpion Venom and Epilepsy

A look at a rare form of epilepsy, Dravet syndrome.

References

  1. https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases
Industry News , Biologically Active Peptides

Robert Brousseau

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Robert Brousseau, the Director of Creative Design and Media at Peptides International, has a wide range of responsibilities that include maintaining the Peptides International brand integrity and creating and implementing all materials related to marketing the company's product line. Since joining the company in 2004, he has incorporated media that now includes printed material such as product brochures and the long-running PEPNET newsletter, along with the company Website, social media, email and other outlets.  Additionally, he assists the staff with IT issues they encounter.  Bob has a background in graphic design, marketing, and advertising, in addition to project management and packaging.  He received his B.A. in Art from the University of Louisville, with additional post-graduate studies in education and design.