Navigation Links
Assemblyman Levine Presents Resolution Recognizing Rare Disease Day at Capitol

Sacramento (PRWEB) February 28, 2013

Today at the State Capitol, Assemblyman Marc Levine (D-San Rafael), in partnership with the California Healthcare Institute (CHI), presented a House resolution recognizing Feb. 28, 2013 as Rare Disease Day.

“On this 30th anniversary of the Orphan Drug Act, we recognize the need for continued incentives for biomedical innovators to bring much needed treatments to patients in need,” Levine said.

It is estimated that there are 7,000 diseases considered rare in the U.S. affecting almost 30 million Americans or about 1 in 10 people. Most rare and ultra-rare diseases have no treatment.

In 1983, Congress enacted the Orphan Drug Act to provide incentives for medical pharmaceutical and product developers to focus on treatments for victims of rare diseases. Since that time, there have been more than 2,700 products studied as possible treatments for rare diseases, and more than 400 of those have been approved by the U.S. Food and Drug Administration. Most of these were for diseases that had no approved treatment.

A disease is considered rare in the U.S. if it affects fewer than 200,000 people. Approximately 50 percent of the people affected by rare diseases are children. Even more prominent are ultra-rare diseases that affect only a few thousand individuals.

Patients with rare diseases and ultra-rare diseases are frequently misdiagnosed or are undiagnosed. Few drug companies conduct research into rare diseases since it is difficult to recover the costs of developing treatments for small, geographically dispersed populations.

BioMarin Pharmaceuticals Inc., based in Novato, Calif., is a pioneer in the area of rare disease research. Dedicated to having a meaningful impact in the lives of underserved patients suffering from diseases that are often ignored, BioMarin has successfully brought four therapies for rare genetic diseases to market. The company has five additional therapies in various phases of clinical trials and multiple pre-clinical programs devoted to treating additional rare diseases. Due to their global success of providing effective therapies, BioMarin has nearly doubled their number of employees in the last five years.

“CHI encourages research and development efforts dedicated to advancing new treatments for patients suffering from rare diseases,” said CHI President and CEO David L. Gollaher, Ph.D. “Rare Disease Day serves as a reminder that while millions of patients have benefited from new treatments, many more remain in need of timely and accurate diagnosis, along with adequate treatment options.”

About CHI
CHI represents more than 275 leading biotechnology, medical device, diagnostics, and pharmaceutical companies, and public and private academic biomedical research organizations. CHI’s mission is to advance responsible public policies that foster medical innovation and promote scientific discovery. CHI’s website is Follow us on Twitter @calhealthcare, Facebook, LinkedIn and YouTube.

Read the full story at

Source: PRWeb
Copyright©2012 Vocus, Inc.
All rights reserved

Related biology technology :

1. ChemoCentryx Presents Positive Results for Novel CCR2 Inhibitor in Preclinical Models of Diabetic Nephropathy at the 44th Annual Meeting of the American Society of Nephrology
2. Tengion Presents New Scientific Data in Presentations at TERMIS North America Annual Meeting
3. Amgen Presents Final Results from the Largest Phase 3 Open-Label Study Assessing the Safety and Efficacy of Nplate® (Romiplostim)
4. 23andMe Presents Top Ten Most Interesting Genetic Findings of 2011
5. Genomic Health Presents Results From First Clinical Decision Making Study of Oncotype DX® Colon Cancer at the 2012 Gastrointestinal Cancers Symposium
6. GeoVax Labs Presents New Findings on Potential HIV Vaccine at 2012 Conference on Retroviruses and Opportunistic Infections (CROI)
7. Fusion presents low proliferation risk, experts conclude
8. ODT Forum Presents Regenerative Technologies
9. PharmAthene Presents Update on SparVax™ rPA Anthrax Vaccine Program at the 2012 World Vaccine Congress
10. Cardium Presents First Quarter 2012 Financial Results and Recent Developments
11. Exosome Diagnostics Presents Data at American Urology Association Demonstrating Potential Utility of Urine Exosomes to Non-invasively Detect and Manage Prostate Cancer
Post Your Comments:
(Date:10/13/2015)... ... 2015 , ... The Pistoia Alliance has appointed Dr Steve ... drive to increase the organization’s impact on the global innovation landscape in life ... its work in supporting pre-competitive collaboration in the life sciences sector. Steve brings ...
(Date:10/13/2015)... Applied BioMath ( ), a computational ... the drug invention process, announced that it will sponsor ... 20-22, 2015 in Boston , MA.  ... CEO of Applied BioMath, will present "QSP approaches enabling quantitative ... trials" on Wednesday October 21st 2015 at 12:30PM.  ...
(Date:10/13/2015)... ARRY ), Avanex Life Sciences ... ), and Progenix Pharmaceuticals, Inc. (NASDAQ: PGNX ) ... (OTCQX: AVXLD), Juno Therapeutics, Inc. (NASDAQ: JUNO ), ... --> Cooperative research and development efforts with the ... to advancements in the treatment of severe wounds.  Biotech ...
(Date:10/13/2015)... and SAN DIEGO , ... Mast Therapeutics, Inc. have agreed to collaborate in the ... poloxamer 188 NF, marketed by BASF under the Kolliphor ... a variety of pharmaceutical and biological applications, such as ... is the starting material for Mast,s lead product candidate. ...
Breaking Biology Technology:
(Date:9/30/2015)... Sept. 30, 2015  With nearly 300,000 Americans living ... new SCIs estimated to reach 12,500 annually, the role ... Resource Services for Independent Living (SCRS-IL) is increasingly ... California opening doors to independence for ... and services, notably assistive technology services and education. ...
(Date:9/29/2015)... Calif. , Sept. 29, 2015 ... improves employee productivity while also saving energy , ... such as Low Power Active Mode and embedded Fujitsu ... transformation Fujitsu today shows that good ... new and refreshed models to its enterprise desktop and ...
(Date:9/28/2015)... 28, 2015  The monitoring of vital signs, ... temperature, is an essential component of patient assessment. ... deterioration in a patient,s condition. However, in general ... typically taken during routine observation rounds only once ... deteriorates between these observation rounds, the warning signs ...
Breaking Biology News(10 mins):