Where's Emil?

World Orphan Drug Congress USA
April 13, 2011 - Washington, DC
The World Orphan Drug Congress USA is North America’s first industry-led orphan drug conference. Dr. Kakkis will give the keynote address. For more information go to
http://www.terrapinn.com/2011/orphandrugusa/

FDA/NIH Spinal Muscular Atrophy Biomarker Qualification Workshop
May 13, 2011 – Silver Springs, MD
The purpose of this SMA biomarker qualification workshop is to explore a variety of molecular and other potential biomarkers for SMA that may be of use in future clinical trials, highlighting the current state of knowledge and the important knowledge gaps regarding each that should be addressed to strengthen their value. Discussion will also include an overview of the process for biomarker qualification at FDA and regulatory candidates for qualification. Dr. Kakkis will be attending. For more info go to
http://www.treat-nmd.eu/events/242/

Rare Disease Workshop Series: Improving the Clinical Development Process
Workshop #2, "Clinical Evaluation of Rare Disease Treatments"
June 14-15, 2011 – Washington, DC
This is the second workshop in a series devoted to improving the clinical development strategies and studies for rare disease treatments. It is supported by the work of the EveryLife Foundation for Rare Diseases’s Scientific Advisory Committee. Rare disease leaders from FDA, academia, NIH and industry will be attending. For more information go to
http://www.kakkis.org/workshopseries/index.html

CBI's Rare Disease Leadership Summit
July 12-13, 2011 – Washington, DC
CBI is proud to present its 6^th Annual Rare Disease Leadership Summit: "Ensuring Access to Orphan Drug Therapies through Collaboration Efforts with Government Researches and Patient Groups" Click here for more info. Dr. Kakkis will be presenting.

26^th Annual National MD/PhD Student Conference
July 17, 2011- University of Colorado, Keystone, CO
Dr. Kakkis will be one of the career panelists where he will share his successes and lessons learned along his career path. The goal is to disseminate practical advice while inspiring participants to find innovative ways to integrate medicine and science.

Orphan Drug Summit 2011
Sept 14-16, 2011- Copenhagen
Dr. Kakkis will be speaking. For more information go to
http://www.orphandrugssummit.com/

Past Events:

FDA Public Advisory Committee Meeting on Orphan Drugs
March 2, 2011 – Dallas, TX
The Advisory Committee for Pharmaceutical Science and Clinical Pharmacology (ACPS-CP) discussed innovative approaches to the development of drugs for orphan and rare diseases to support decisions such as dose and trial design selection. Dr. Kakkis spoke during the public session.
http://www.fda.gov/AdvisoryCommittees/Calendar/ucm240582.htm

RDLA Meeting: Current Budget Outlook
March 1, 2011 – Washington, DC
Representatives from the Coalition for Health Funding, Federation of American Societies for Experimental Biology and Alliance for a Stronger FDA spoke about the current budget outlook. Foundation staff attended. You can listen to the RDLA meeting by Streaming recording or Download recording.

Cocktail Reception and Movie Screening of Extraordinary Measures
February 28, 2011 – Washington, DC
Rare Disease Legislative Advocates (RDLA) hosted a cocktail reception and movie screening of Extraordinary Measures in DC. Special guests John Crowley, the father who inspired the movie and former Congress Member Dick Gephardt attended along with Foundation staff.

2^nd Annual SBMRI Rare Disease Symposium
February 25, 2011 – San Diego, CA
Sanford-Burnham Medical Research Institute hosted its 2^nd Annual Rare Disease Symposium in San Diego. Scientists and rare disease group advocates from around the country gathered to discuss the newest findings and the future of rare disease research. Dr. Kakkis presented on "Developing Treatments for Rare Diseases: Overcoming the challenges to translation to human use." View the Webcast

7^th Annual WORLD Symposium
February 16-18, 2011 - Las Vegas NV
Dr. Kakkis spoke on “Update on Improving the Development Process for Rare Diseases.” For more information go to http://www.lysosomaldiseasenetwork.org/

Rare Disease Caucus Congressional Staffer Briefing
February 1, 2011 – Washington DC
Julia Jenkins, the Foundation’s Director of Government Relations, John Crowley, CEO of Amicus Therapeutics and father of two children with Pompe, the Muscular Dystrophy Association and the Cystic Fibrosis Foundation discussed the current state of rare diseases and potential opportunities for improvement with staffers from Energy and Commerce Chairman Upton (R-MI), Rep Crowley (D-NY) and Rep. Lance (R-NJ) Offices.

CureDuchenne 2nd Annual Scientific Summit
January 27-29 - Newport Beach, CA
The Duchenne Summit brought together top Duchenne scientists, biotech executives, regulatory experts and physicians from around the country to develop research strategy for the coming year. For more information go to http://www.cureduchenne.org/site/Calendar?id=100082&view=Detail

2^nd Annual Legends of Golf Tournament & Dinner
Jan. 9, 2011 – San Francisco, CA
Co hosted by the Children’s Rare Disease Network, the National MPS Society, CureDuchenne Muscular Dystrophy, Global Genes Project and the Ryan Foundation. The Legends of Golf Tournament brought together executives from the biotech industry and financial community for an intimate event to raise funds for the EveryLife Foundation for Rare Diseases to help accelerate treatments for rare diseases. Pfizer, Burrill and Company, West Wind Wines, Amicus Therapeutics, Fold RX, Eugene Son and Family, and Raptor were sponsors. Check out event photos at www.kakkis.org/golf

World Orphan Drug Congress
Nov. 29 - Dec. 1, 2010 – Geneva, Switzerland
The World Orphan Drug Congress is the World’s first industry-led Orphan Drug conference tackling global trends, market access and Big Pharma impact in rare diseases from GlaxoSmithKline, Pfizer and others. Dr. Kakkis presented. http://www.terrapinn.com/2010/orphandrugs/index.stm

NORD's Fall Corporate Council Meeting
November 17, 2010 – Washington, DC
EveryLife staff attended the meeting along with senior executives from more than 70 leading companies and organizations.

Prescription Drug User Fee Act (PDUFA) Reauthorization Stakeholder Meeting
November 17, 2010 – Washington, DC
The Foundation is participating as a stakeholder for the Prescription Drug User Fee Act Reauthorization process. The meetings called by the FDA include patient and consumer advocacy groups, health care professionals, and scientific and academic experts. The statutory authority for PDUFA expires in September 2012. At that time, new legislation will be required for FDA to continue collecting user fees for the prescription drug program. Currently, orphan designated drugs are exempt from these fees. For more information go to: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm117890.htm

NORD Board of Directors Meeting
November 16, 2010 - Washington DC
Dr. Kakkis attended the meeting.

Rare & Neglected Disease Congressional Caucus Planning Meeting
November 15, 2010 – Washington DC
Rare Disease Legislative Advocates group hosted a planning meeting at FasterCures for the newly formed Rare Disease Caucus. John Crowley, whose leadership helped secure the Caucus co-chairs, facilitated the meeting. Crowley has two children with Pompe, a neuromuscular disease and was the inspiration for the film "Extraordinary Measures." The goal of the planning session was to receive input from the whole rare and neglected disease community and build consensus on a broad and inclusive federal legislative agenda to advance multiple initiatives to accelerate the development of new treatments for rare and neglected diseases. EveryLife Staff helped organize the meeting. More than 60 patient organizations participated.

Translational Research Priorities for Infantile (CLN1) and Late Infantile (CLN2) Forms of Batten Disease
Nov. 11-12, 2010 - Bethesda, MD
Dr. Kakkis presented.

Rare Disease Workshop Series: Improving the Clinical Development Process
Workshop 1: Optimizing the Choice of Statistical Analysis and Study Design for Ultra Rare Diseases
November 8, 2010 – Bethesda, MD
This was the first workshop in a series devoted to improving the clinical development strategies and studies for rare disease treatments. It is supported by the work of the EveryLife Foundation for Rare Diseases’s Scientific Advisory Committee. Tom Fleming, Ph.D., Professor of Biostatistics at the University of Washington was the keynote speaker. A small group of interested leaders from FDA, academia, NIH and industry participated. For more information go to www.kakkis.org/workshop

Prescription Drug User Fee Act (PDUFA) Stakeholder Meeting
October 22, 2010 – Washington, DC (via Conference Call)
The FDA has been holding regular stakeholder meetings for patient groups regarding the next reauthorization of PDUFA(V). The Foundation submitted the three campaign goals on behalf our 165 partners to be included in the PDUFA goals as well, advocated for rare diseases to be a priority. For more information go to: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm117890.htm

6^th Modern Drug Discovery & Development Summit
October 21, 2010 – San Francisco, CAs
Sponsored by GTCbio. EveryLife staff attended the 4th Biomarker Discovery and Development track to help assist with the planning of the Rare Disease Workshop Series. http://www.gtcbio.com/userconference.aspx

FIGON Dutch Medicine Days
Oct. 4-6, 2010 - Netherlands
Dr. Kakkis was a keynote speaker. “No Disease is Too Rare to Deserve Treatment.” http://www.figondmd2010.nl/.