ACTION ALERT: Costs of Caring for Children with Rare Disease Survey
Shivani B. Nazareth, RARE Blog contributor and a board-certified genetic counselor with Counsyl, Inc, is conducting an anonymous survey to better understand the financial cost of caring for a child with a rare genetic disease. By participating in this survey, you will contribute to a research paper that will quantify the financial burdens endured by parents of [...]
75 Days Until World Rare Disease Day!
It’s December 15th and that means only 75 days left until World Rare Disease Day on February 29th, 2012! The number 75 is significant for the rare community. According to the NIH’s Office of Rare Disease Research, 75% of RARE diseases affect children making this disease category one of the most deadly and debilitating for [...]
Batten Disease Awareness Weekend Bringing Hope for Children with a Fatal Disease—and No Cure in Sight June 3-5, 2011
Batten Disease Awareness Weekend Bringing Hope for Children with a Fatal Disease—and No Cure in Sight June 3-5, 2011 June 1, 2011 (Reynoldsburg, OH) – Batten Disease Support and Research Association (BDSRA) is holding its 3rd annual Batten Disease Awareness Weekend, June 3-5, 2011 to bring recognition to a rare but devastating disease. Batten disease [...]
PhRMA Urges Congress to Reauthorize Legislation for Pediatric Drugs
May 10, 2011 By: Patricia Van Arnum BioPharm Bulletin The Pharmaceutical Research and Manufacturers of America (PhRMA) urged Congress to reauthorize two pieces of legislation relating to pediatric studies and exclusivity, the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA). PhRMA made the comments in testimony in a public meeting [...]
Genetic Diseases In Children (GDC) Conference – A Grand Slam!
The Wadsworth Center, March 7-10, NYC at, was host to the Genetic Diseases In Children Conference. My recommendation . . . make it to the meeting next year! Here’s why! Most conferences discuss theories, postulate and talk a lot about ‘what can and should be done’ about a topic. The GDC Conference created an agenda [...]
GLOBAL GENES PROJECT ANNOUNCES “WEAR THAT YOU CARE™” DENIM AWARENESS CAMPAIGN FOR RARE DISEASE DAY 2011
Statistics Show 95% of Rare Diseases Have No FDA Approved Drug Treatments; Only 352 New Drugs Developed and Approved For Tens of Millions of People Since 1983 DANA POINT, Calif. – January 26, 2011 – Monday, February 28, 2011, is the 4th annual Rare Disease Day — a day when people worldwide will show their [...]
Another Global Pharmaceutical Company Jumping In To Help Rare Disease Community!
More news from Pharma and the giant Glaxo Smith Kline about new division focusing on rare disease – It seems that over the last 4 months we have heard about various Pharma organizations with new commitments to rare disease, focusing money and research to help find treatments and cures for some of the rarest conditions. [...]
Families look to orphan drug development at University of Minnesota
Orphan diseases affect 25 million people in the United States. By Taylor Selcke, MN Daily.com For Michael Zimanske, years of holidays and birthdays spent in hospitals began after a routine kindergarten check-up. The doctor said he wasn’t growing like a normal 5-year-old, and full body X-rays revealed the abnormalities in his joints. It took two [...]
Senator Brownback Introduces the Creating Hope Act of 2010; Bill Would Change the Priority Review Voucher Program and Extend it to Applications for Rare Pediatric Diseases
By Kurt R. Karst – FDA Law Blog Hyman, Phelps & McNamara, P.C. Yesterday, Senator Sam Brownback (R-KS) (along with Sen. Sherrod Brown (D-OH)) introduced S. 3697, the Creating Hope Act of 2010. The bill would amend FDC Act § 524 to change the transferable Priority Review Voucher (“PRV”) program created by the 2007 FDA [...]
FDA approval of Geron’s Embryonic Stem Cell Trial Has Widespread Implications For Rare Disease
In its Friday press release, Geron, the California-based biotech, announced the FDA’s clearance to proceed with its embryonic stem cell trial for spinal cord injury. This will be the first clinical trial to test the use of embryonic stem cells in human patients, and it will have widespread implications for many other common and rare diseases, including Canavan disease.