Where’s Emil?

Visiting Lecturer at Shire HGT
Boston, MA - March 4, 2010
Dr. Kakkis will be speaking about "Efficient Regulatory Strategies for Rare & Orphan Diseases." The Visiting Lecturer program is housed under Global Clinical and Medical Affairs. The objectives are to provide education and information on rare genetic disorders; establish and emphasize the role that experimental research and practice play with treatments for rare genetic disorders; encourage instruction in the field of rare genetic disorders; create an awareness of the research opportunities within the field of rare genetic disorders; and provide information and showcase their (Lecturers’) research to the Shire employee community and stimulate an interactive discussion amongst the various attendees.

Center for Business Intelligence’s West Coast Forum on Early Access Programs
San Francisco, CA – March 10, 2010
Dr. Kakkis will be on a Panel Discussion “Working with Patient Advocacy Groups in the Development of Early Access Programs.” Patient advocacy groups are a key influencer of early access programs. During this session, review advocacy group efforts to assist patients and interface with industry in an effort to guide responsible access. Learn about strategies for working with advocacy groups to develop and implement successful early access programs. Establish effective partnerships with patient groups. For more information go to http://www.cbinet.com/show_conference.cfm?confCode=PC10024

American College of Medical Genetics Annual Clinical Genetics Meeting
Albuquerque, New Mexico – March 25 – 28, 2010
The Kakkis Foundation will be hosting a booth featuring the CureTheProcess Campaign & the Global Genes Project at the annual conference. For more information go to http://www.acmgmeeting.net/acmg10/public/enter.aspx

Society for Inherited Metabolic Disorders 2010 Annual Meeting
Albuquerque, New Mexico – March 28 – 31, 2010
The Kakkis Foundation will be hosting a booth featuring the CureTheProcess Campaign & the Global Genes Project at the annual conference. For more information go to http://www.simd.org/meetings/SIMD2010/

11th International Symposium on Mucopolysaccharide and Related Diseases
South Australia - June 23 – 27, 2010
Dr. Kakkis is a "Friends of MPS Sponsor" and he will be attending. For more information go to http://www.plevin.com.au/mps2010/

Past Events:

WORLD Symposium 2010
Miami, FL - February 10-12, 2010
Dr. Kakkis spoke at the Lysosomal Disease Network’s annual meeting. This year’s theme is "We're Organizing Research on Lysosomal Diseases." Dr. Kakkis discussed Transforming the Development of Treatments for Lysosomal Storage Disorders. http://www.lysosomaldiseasenetwork.org/

Kakkis EveryLife Foundation’s Scientific Advisory Committee Meeting
Washington, DC - February 6, 2010
The Foundation’s Scientific Advisory Committee begun developing the analytical plan for analyzing data from past rare disease clinical trials to help identify and validate more powerful statistical models for traditional study designs and to help devise and test alternative clinical trial designs.

Institute of Medicine’s 3rd Meeting for Accelerating Rare Disease Research and Orphan Product Development
Washington DC - February 4, 2010
Dr. Kakkis spoke to the Committee which was formed in response to a request from the NIH Office of Rare Diseases and the FDA Office of Orphan Products Development. The committee of the IOM will prepare a report that will assess existing strategies to promote research discoveries and development of orphan products to improve the health of people with rare diseases. For more information go to http://www8.nationalacademies.org/cp/projectview.aspx?key=49099

Burrill & Company 10th Annual Reception
San Francisco - January 12, 2010
Dr. Kakkis & Foundation COO John Ditton joined Burrill & Company for their reception for the Annual JP Morgan Healthcare Conference.

Inaugural EveryLife Foundation Legends of Golf Tournament
San Francisco, CA – January 10, 2010
As thousands of investors, bankers, and biotech company executives poured into San Francisco for the Annual JP Morgan Healthcare Conference, the Kakkis EveryLife Foundation hosted its inaugural Legends of Golf Tournament to bring attention to needed regulatory changes that will accelerate biotech innovation for rare diseases and spur investment in the industry. The Golf Tournament was co‐hosted by the Children’s Rare Disease Network, National MPS Society and the Ryan Foundation and was sponsored by the Biotechnology Industry Organization, BioMarin, FoldRx, Michael Lee, RedMile Group and Adams Golf. More than 70 golfers enjoyed 18 holes at the Presidio Golf Course, wine tasting, dinner and some celebrity entertainment including Rodney Dangerfield and Tiger Woods impersonators. For more information go to http://www.kakkis.org/golf

Extraordinary Measures Private Screening.
San Francisco – January 6, 2010
Dr. Kakkis and Wife Dr. Jenny Soriano attended the private screening of Extraordinary Measures in San Francisco hosted by Mayor Gavin Newsom with special guest Harrison Ford. Click here to see the trailer http://extraordinarymeasuresthemovie.com/

7th International and Latin America Congress
Cancun, Mexico – December 6-9, 2009
Dr. Kakkis attended the Congress, hosted by the International Society of Neonatal Screening (ISNS) and the Latin American Society of Inborn Errors of Metabolism (SLEIMP). To find out more go to: http://www.nsiemcancun2009.com

CNS Manifestations of Inborn Errors of Metabolism Workshop (ICMW)
Bethesda, MD – December 7-8, 2009
Dr. Kakkis spoke at the ICMW, a new workshop, jointly sponsored by the National Institute of Health’s (NIH’s) Office of Rare Diseases Research (ORDR) and the Food and Drug Administration’s (FDA’s) Center for Drug Evaluation and Research, Division of Gastroenterology and Inborn Errors of Metabolism Products (DGIP). The goal of the workshop is to bring together those involved in research and investigational treatments for central nervous system (CNS) aspects of inborn errors of metabolism (IEM) and to discuss ways to translate these therapies to clinical trials and beyond. Click here to view the agenda:
http://www.rarediseases.info.nih.gov/Inborn_Errors_Metabolism/ Inborn_Errors_Metabolism_Agenda.pdf

Institute of Medicine Meeting for Accelerating Rare Disease Research and Orphan Product Development
Washington, DC – November 23, 2009
Dr. Kakkis attended the meeting to share the CureTheProcess campaign goals during the public session. http://www8.nationalacademies.org/cp/meetingview.aspx?MeetingId=3666

NORD Executive Board & Corporate Council Meeting
Washington DC – November 11-12, 2009
Dr. Kakkis attended his first meeting as an elected member of NORD’s board and presented to NORD’s Corporate Council.

The Burrill Personalized Medicine Meeting
San Francisco, CA - November 9, 2009
Dr. Kakkis was extended an invitation to join the ‘Expert Luncheon’ table. His topic was: “Personalized Medicine: Analyzing Commons Disorders Like Rare Diseases” http://www.burrillandco.com/personalized_medicine_2009/2009_Agenda.html

California Healthcare Policy Forum “Innovation at a Crossroads”
San Francisco, CA – November 5, 2009
The KELF team attended along with more than 100 leading executives from the science, business and public sectors to hear of the critical opportunities and issues facing the biomedical community. http://www.chi.org/hcpf/default.aspx

Dr. Kakkis Met with Investment Firms
New York, NY - October 27-28, 2009
Dr. Kakkis meet with investment firms in New York in efforts to expand support for the CureTheProcess campaign and explain how it will help accelerate investment in biotechnology.

Patient Access to Investigational Drugs Media Luncheon
Washington, DC - September 30, 2009
Dr. Kakkis joined a panel of stakeholders to discuss the new FDA regulations on compassionate use.
Read BIO World Article

International Lysosomal Disease Meeting
Nagoya, Japan - September 26-28, 2009
Dr. Kakkis presented on the future prospects of Enzyme Replacement Therapy for Lysosomal Disease.

Dr. Kakkis met with Congress Members to Discuss Brownback/Brown Amendment
Washington, DC - September 10, 2009
Dr. Kakkis met with Senators Brownback and Brown’s Offices to thank them for their support of improving FDA regulation for Rare and Neglected Diseases. Read more about it at:
Emil’s Blog
Press Room

Rare Disease Meeting for Legislative Advocates
Washington DC - September 9, 2009
The Kakkis Foundation hosted a meeting for legislative advocates to share information about legislative and policy priorities and goals including a Rare Disease Congressional Caucus and Congressional Hearing. Genetic Alliance, Hemophilia Federation, NORD, National MPS Society, Cystic Fibrosis Association, National PKU Alliance, and National Society of Genetic Counselors all attended.

The Manton Center for Orphan Disease Research at Children's Hospital Boston
Boston MA – September 9, 2009
Dr. Kakkis was the Manton Center’s first visiting scientist. Dr. Kakkis spoke to a group of patients, families, students, and health care providers about the CureTheProcess Campaign. The Manton Center supports Dr. Kakkis's efforts to bring treatment to all patients, regardless of the prevalence of their disorder. http://www.childrenshospital.org/cfapps/research/data_admin/Site2673/mainpageS2673P18.html

International Congress of Inborn Errors of Metabolism Conference
San Diego, CA – September 2, 2009
Dr. Kakkis spoke at the plenary session about the Future Prospects of Enzyme Replacement Therapy. The Foundation sponsored a booth at the event and hosted a roundtable discussion with doctors & scientist from around the world about the CuretheProcess campaign.

CureTheProcess Launch Lunch
Novato CA - August 21, 2009
The Kakkis Foundation hosted a luncheon for its friends and neighbors to share updates and the progress of the CureTheProcess Campaign.

Genetic Alliance Annual Conference: Discovering Openness in Health Systems
Bethesda, MD – July 16–17, 2009
The Kakkis EveryLife Foundation sponsored an information booth at the 2009 Genetic Alliance. Speakers included FDA officials as well the newly appointed NIH director Dr. Francis Collins. You can find additional speakers and more information about the Conference at: http://www.geneticalliance.org/conference09.

Chamber of Commerce discussion on Orphan Drug Policy
Washington D.C. – June 4, 2009
Government Relations Director, Julia Jenkins reported that the Social Security Administration Commissioner shared a similar perspective of the CURETHEPROCESS campaign. Echoing the call for process improvements targeting rare disease treatments, Commissioner Michael Astrue was quoted as saying “FDA's new leadership should focus on improving the regulatory predictability for orphan drug development”. To view the webcast please visit: http://ncf.uschamber.com/orphan_drugs/

18th Annual Alpha One National Education Conference
San Francisco, CA – June 5, 2009
Patient Advocacy Liaison, Steve Smith joined over 450 attendees at the Annual National Education Conference, to share the CURETHEPROCESS Campaign goals with partner patient organizations. For more information about the conference go to: http://www.alpha1.org/education/nateduconf.php.

NORD’S Partners in Progress Summit
Washington DC - May 14, 2009
Dr. Kakkis presented his experiences in rare disease drug development and reasons for the CURETHEPROCESS campaign. The Summit was part of an ongoing collaborative effort with the National Organization of Rare Diseases (NORD) as they create a national agenda for the Rare Disease Community.
Watch Dr. Kakkis’ Presentation
Watch NORD Video Recap
Read Summit Report