National Tay-Sachs and Allied Diseases Association – Spring 2013 Newsletter Over 300 of NTSAD’s friends and family members gathered in Boston on November 1 and in the New York area on December 5 to celebrate NTSAD’s 55th Anniversary. Former CEO of Genzyme, Henri Termeer, was honored in Boston for his pioneer role in bringing treatments [...]
Parent Project Muscular Dystrophy (PPMD) published a white paper, Putting Patients First – Recommendations to speed access to new therapies for Duchenne muscular dystrophy and other rare, serious and life-threatening neurologic disorders. Emil Kakkis served on the paper’s Advisory Committee along with Richard Finkel, MD, Marlene E. Haffners, MD, MPH, Laurie Letvak, MD, H. Lee. [...]
Lexington, MA – April 9, 2013 Shire plc (LSE: SHP, NASDAQ: SHPG) today launched a Rare Disease Impact Report, which uncovers the health, psycho-social, and economic impact of rare diseases on patient and medical communities in the United States (US) and United Kingdom (UK). The report, developed in collaboration with an external advisory board of [...]
INSIDE WASHINGTON’S FDA WEEK – March 8, 2013 Rare disease stakeholders are joining forces to write a white paper they hope FDA can use as part of its forthcoming accelerated approval guide, which is due later this year under an FDA Safety and Innovation Act mandate. The effort, although not spurred by the current fiscal [...]
From the FDA Law Blog Posted: 15 Jan 2013 07:07 PM PST By Alexander J. Varond FDA announced earlier this week that it will hold a public hearing to obtain input on a potential new pathway to expedite the development of drugs and biological products for serious or life-threatening conditions that would address an unmet [...]
The potential automatic spending cuts, or sequestration, scheduled to take effect in January 2013, will be devastating for the future U.S. leadership of research and development and would delay access to new medical treatments. Sequestration would slash federal investments in critical health, scientific, medical and biological research aimed at discovering treatments, moving safe and effective [...]
Several special Rare Disease events are scheduled for November 13-15 in Washington DC. Visit rareadvocates.org to see the list and schedule. events include: Alliance for Stonger FDA: Election ReCap and Annual Meeting Global Genes | Rare Project MeetUp™ Friends-Brookings Conference on Clinical Cancer Research Rare Disease Congressional Caucus Briefing RareVoice Awards Gala Rare Disease Series [...]
Bay Area Science Festival November 1, 2012 at 6pm Roxie Theater 3117 16th Street, San Francisco Free Admission, Q&A with filmmakers & Heather Kirkwood to follow screening. RARE, a documentary about an extraordinary mother who mobilizes research that could help her daughter and others with a rare genetic condition. www.rarefilm.org Filmed with intimate access over [...]
Celebrities and Rare Disease Community Turn Out To Support Global Genes Project’s 1st Annual “Tribute to Champions of Hope” Benefit (via PR Newswire) Rare Disease Industry Leaders and Advocates Honored; Recording Artists Chris Mann, Katrina Parker and Elliott Yamin Gave Touching Performances DANA POINT, Calif., Oct. 2, 2012 /PRNewswire-USNewswire/ – On September 27, 2012, rare disease patients, [...]
On November 14, 2012, Rare Disease Legislative Advocates will host its inaugural RareVoice Awards Gala to honor and thank Members of Congress, Congressional Staff and Patient Advocates for their advocacy to ensure The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187 included provisions that spur the development of life saving treatments for rare disease patients. FDASIA [...]
