Creating Awareness Of Rare Diseases & Rare Disease Advocacy Day (March 1, 2012)

From the 6 December 2011 article at Medical News Today

“Rare diseases”, by their very definition, occur in no more than 5 people out of every 10,000 inhabitants. Barely noticed by the general public, only around 1,000 of the currently 6,000 or so different rare diseases currently listed on the Internet platform Orphanet are treatable nowadays. “And only a very small number are curable,” says Till Voigtländer from the Clinical Institute of Neurology at the MedUni Vienna and an expert on rare diseases.

80 per cent of rare diseases have a genetic origin, with the remaining 20 per cent being caused by diseases of the immune system, infections or poisoning. Clinically, rare diseases are frequently characterised by a severe, chronic progression of the disease and/or a shortened life expectancy.

So it is all the more important to create awareness of this type of disease, for example at the Austrian Congress on Rare Diseases 2011 at the MedUni Vienna, which is being held on the 2nd and 3rd of December and arranged jointly by Till Voigtländer and Reginald Bittner from the Centre for Anatomy and Cell Biology…..

 

Helping Rare Disease Patients Find Their Voice

(with information on Rare Disease Advocacy Day, March 1, 2012

Patients often need advocates, and that can be especially true for people with a rare disease, who have unique problems and may have little or no support or available treatment.

To help them become advocates for themselves and others with their disease, the Food and Drug Administration (FDA) is sponsoring its first “FDA Rare Disease Patient Advocacy Day” on March 1, 2012.

This event at FDA headquarters in Silver Spring, Md., commemorates the fifth annual Rare Disease Day, a global campaign to raise awareness of the more than 250 million people worldwide who suffer from rare diseases. Some of these diseases have familiar names—such as cystic fibrosis and Lou Gehrig’s disease—but there are thousands of others whose name is only known to those affected by them.

The Patient Advocacy Day sessions—some of which will also be webcast—are partially designed to increase awareness within the rare disease community of FDA’s roles and responsibilities in the development of medical products for the diagnosis, prevention, and/or treatment of rare diseases or conditions. (Click here for the registration form.)

Related articles

• We’ve Come A Long, Long Way Together… But We’re Not There Yet! (spittoon.23andme.com)
• Spread the Word: Rare Disease Day 2012 (rainintorainbows.com)
• ‘LOVE and LUCK for Rare Diseases’ Campaign and ‘I Challenge Rob’ Contest to enable new programs for Rare Diseases (prweb.com)
• Rare Disease Day Is February 29, 2012 (cmtnyus.wordpress.com)

A safer, more effective morphine may soon be possible (& drug information resources)

A safer, more effective morphine may soon be possible

From the March 24 2011 Science Daily news item

ScienceDaily (Mar. 25, 2011) — An orphan drug** originally used for HIV treatment has been found to short-circuit the process that results in additional sensitivity and pain from opioid use. The study by researchers at the Indiana University School of Medicine is reported in the March 25, 2011 issue of Brain, Behavior and Immunity***….

** Information on this orphan drug, AMD3100, may be found  through Drug Information Portal (US National Library of Medicine) at the Plerixafor [USAN] Web page

For related drug information resources, please see my previous posting Drug Information Product…

 

***For suggestions on how to get this article for free or at low cost, click here

 

Related Articles

• Orphan Products: Hope for People With Rare Diseases
• FDA basics – Orphan Drugs
• Developing Orphan Products: FDA and Rare Disease Day(Food and Drug Administration)
• New drug restores responsiveness to morphine (news.bioscholar.com)
• Pain-killing drug more potent, longer lasting than morphine: Study (news.bioscholar.com)
• Prison term for Snohomish nurse who watered down morphine (seattlepi.com)