The Good, The Bad and The Ugly: How to Approach Unsolicited Advice and Unexpected Actions That Made Your Day

Moderator Biography

Tiffany House

President, Acid Maltase Deficiency Association; Chairman/Board Member, International Pompe Association

Tiffany House is President, Acid Maltase Deficiency Association (AMDA), Inc., San Antonio, Texas; a non-profit advocacy organization for patients with Pompe Disease. Hosts/spearheads biannual conference, communicates with industry, current on latest advancements in treatment and patient care, attends various scientific/patient conferences, and is Chair or member of various Working Groups or Committees.

Chairman/Board Member, International Pompe Association (IPA), Baarn, the Netherlands; an international Pompe patient support organization. The mission of the IPA is to “Campaign for early diagnosis and effective, affordable and safe therapies. Strive to provide information and support to all patients, their families and others with interests in Pompe Disease.” Board Member; participates in regular conference calls with Board and industry, annual meetings, and face-to-face meetings with industry and scientific/medical experts.

Active/Voting Member, Lysosomal Disease Network (LDN) External Advisory Committee (EAC), National Institutes of Health (NIH) Rare Diseases Clinical Research Network (RDCRN), University of Minnesota, Minneapolis, Minnesota; participates in annual meetings and communications, evaluates Pilot Project proposals and Fellowship applications, reviews clinical research and opportunities with a focus on MPS, Pompe, and Fabry diseases and two cores: administrative and education.

Member, Lysosomal Storage Disorders (LSD) Advocacy Coalition; participates in monthly meetings to work together on state and federal issues such as Medicare Home Infusion, medical research and Newborn Screening as well as state and federal issues.

Panelist, Bayer Pompe Lived Experience Expert (LEE) Standing Expert Panel (SEP); collaborates with a broad geographical spread of experts to obtain ongoing deep insights into the disease characteristics, patients unmet needs and preferences, the current standard treatments, and the potential impact of novel therapies.

Newborn Screening Advisory Committee member.

Patient Representative/Voting Member of Four Advisory Committees (AC), Food and Drug Administration (FDA), Washington, D.C.; serves as the “voice of the patient.” Active team member reviewing Lumizyme (Pompe treatment). Committed to email correspondence, teleconferences, and in-person meetings in D.C. Lumizyme AC Voting Member, evaluated application with recommendations, October 2008. Patient Representative Role guest speaker, FDA Patient Representative workshops, 2009 and 2010.

Patient Representative, Niemann-Pick Type C and NAGS Deficiency AC, January 2010; Patient Representative, Direct-to-Consumer Genetic Testing AC, March 2011.

Tiffany received her Juris Doctorate (Suma Cum Laude) in 2011 at St. Mary’s University School of Law, San Antonio, Texas and an M.A. in English, 2007 and B.A. in English, 2005 (Thesis: Pompe’s Disease: My Experiences and the Search for a Cure) at the University of Texas at San Antonio, Texas.