Survey for Penn Center for Orphan Disease Research & Therapy

Orphan diseases represent a collection of disorders that afflict <200,000 individuals for any single disease type, and yet, as there are ~7,000 orphan diseases, in aggregate over 25 million US citizens suffer substantial morbidity and mortality.

The Center for Orphan Disease Research and Therapy would like to find out types of Orphan Disease Research being conducted at Penn and CHOP.

Please fill out the survey as accurately as possible to gain access to the Center for Orphan Disease Research and Therapy for Funding initiatives, Resources and Research links.

This SURVEY is AVAILABLE to ALL Penn and CHOP Faculty and is REQUIRED for MEMBERSHIP in the Center. There are a total of 16 questions and the survey should take no longer than 15 min. to complete.

Page 1 of 2 - Questions 1-10-Information about you & your Orphan Disease Research.

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1. Name 1. Name First Middle Last
2. Degree

2. Degree MD Ph.D MD PHD Other Other
3. Email Address:
4. Identify your primary departmental appointment in the PSOM.
5. Non PSOM, where is your primary appointment?

5. Non PSOM, where is your primary appointment? CHOP School of Arts and Sciences School of Dental Medicine School of Engineering and Applied Science School of Nursing School of Veterinary Medicine Other Other
5a. Non PSOM, please identify your department.
6. Categories of Rare Diseases that you are studying (please check all that apply) :

6. Categories of Rare Diseases that you are studying (please check all that apply) : Autoimmune/Immune Disorders Blood Disorders Cancer Cardiovascular Diseases Gastrointestinal Disorders Genetic Disorders Growth and/or Developmental Disorders Infectious Diseases Metabolic Disorders Musculoskeletal Disorders Neurological and/or Neurodegenerative Disorders Ocular Disorders Renal Disorders Respiratory Disorders Transplantation Other (please Specify) Other (please Specify)
7. Based on your amount of effort, please name the Primary Rare Disease you are studying:

Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your Primary Rare Disease Studies:
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical
Primary Rare Disease	Primary Rare DiseaseAnimal Models	Primary Rare DiseaseCellular/ Molecular	Primary Rare DiseaseOutcomes/ Comparative Effectiveness	Primary Rare DiseaseCellular Models	Primary Rare DiseaseClinical Patient Care	Primary Rare DiseaseClinical Trials	Primary Rare DiseasePre- Clinical

Do you study other Rare Diseases?

Do you study other Rare Diseases? Yes No
In order of effort, please name SECOND Rare Disease you are currently studying:

Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your SECOND Rare Disease Studies:
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical	Other
Second Rare Disease	Second Rare Disease Animal Models	Second Rare Disease Cellular/ Molecular	Second Rare Disease Outcomes/ Comparative Effectiveness	Second Rare Disease Cellular Models	Second Rare Disease Clinical Patient Care	Second Rare Disease Clinical Trials	Second Rare Disease Pre- Clinical	Second Rare Disease Other

Do you study more than TWO Rare Diseases?

Do you study more than TWO Rare Diseases? Yes No
In order of effort, please name THIRD Rare Disease you are currently studying:

Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your THIRD Rare Disease Studies:
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical	Other
Third Rare Disease	Third Rare DiseaseAnimal Models	Third Rare DiseaseCellular/ Molecular	Third Rare DiseaseOutcomes/ Comparative Effectiveness	Third Rare DiseaseCellular Models	Third Rare DiseaseClinical Patient Care	Third Rare DiseaseClinical Trials	Third Rare DiseasePre- Clinical	Third Rare DiseaseOther

Do you study more than THREE Rare Diseases?

Do you study more than THREE Rare Diseases? Yes No
In order of effort, please name FOURTH Rare Disease you are currently studying:

Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your FOURTH Rare Disease Studies:
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical	Other
Forth Rare Disease	Forth Rare DiseaseAnimal Models	Forth Rare DiseaseCellular/ Molecular	Forth Rare DiseaseOutcomes/ Comparative Effectiveness	Forth Rare DiseaseCellular Models	Forth Rare DiseaseClinical Patient Care	Forth Rare DiseaseClinical Trials	Forth Rare DiseasePre- Clinical	Forth Rare DiseaseOther

Do you study more than FOUR Rare Diseases?

Do you study more than FOUR Rare Diseases?
Yes No
In order of effort, please name FIFTH Rare Disease you are currently studying:

Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your FIFTH Rare Disease Studies:
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre-Clinical	Other
Fifth Rare Disease	Fifth Rare DiseaseAnimal Models	Fifth Rare DiseaseCellular/ Molecular	Fifth Rare DiseaseOutcomes/ Comparative Effectiveness	Fifth Rare DiseaseCellular Models	Fifth Rare DiseaseClinical Patient Care	Fifth Rare DiseaseClinical Trials	Fifth Rare DiseasePre-Clinical	Fifth Rare DiseaseOther

Do you study more than FIVE Rare Diseases?

Do you study more than FIVE Rare Diseases? Yes No
In order of effort, please name SIXTH Rare Disease you are currently studying:
Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your SIXTH Rare Disease Studies
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical	Other
Sixth Rare Disease	Sixth Rare DiseaseAnimal Models	Sixth Rare DiseaseCellular/ Molecular	Sixth Rare DiseaseOutcomes/ Comparative Effectiveness	Sixth Rare DiseaseCellular Models	Sixth Rare DiseaseClinical Patient Care	Sixth Rare DiseaseClinical Trials	Sixth Rare DiseasePre- Clinical	Sixth Rare DiseaseOther

Do you study more than SIX Rare Diseases?

Do you study more than SIX Rare Diseases?
Yes No
In order of effort, please name SEVENTH Rare Disease you are currently studying:
Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your SEVENTH Rare Disease Studies
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical	Other
Seventh Rare Disease	Seventh Rare DiseaseAnimal Models	Seventh Rare DiseaseCellular/ Molecular	Seventh Rare DiseaseOutcomes/ Comparative Effectiveness	Seventh Rare DiseaseCellular Models	Seventh Rare DiseaseClinical Patient Care	Seventh Rare DiseaseClinical Trials	Seventh Rare DiseasePre- Clinical	Seventh Rare DiseaseOther

Do you study more than SEVEN Rare Diseases?

Do you study more than SEVEN Rare Diseases? Yes No
In order of effort, please name EIGHTH Rare Disease you are currently studying:
Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your EIGHTH Rare Disease Studies
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical	Other
Eighth Rare Disease	Eighth Rare DiseaseAnimal Models	Eighth Rare DiseaseCellular/ Molecular	Eighth Rare DiseaseOutcomes/ Comparative Effectiveness	Eighth Rare DiseaseCellular Models	Eighth Rare DiseaseClinical Patient Care	Eighth Rare DiseaseClinical Trials	Eighth Rare DiseasePre- Clinical	Eighth Rare DiseaseOther

Do you study more than EIGHT Rare Diseases?

Do you study more than EIGHT Rare Diseases? Yes No
In order of effort, please name NINTH Rare Disease you are currently studying:
Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your NINTH Rare Disease Studies
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical	Other
Nineth Rare Disease	Nineth Rare DiseaseAnimal Models	Nineth Rare DiseaseCellular/ Molecular	Nineth Rare DiseaseOutcomes/ Comparative Effectiveness	Nineth Rare DiseaseCellular Models	Nineth Rare DiseaseClinical Patient Care	Nineth Rare DiseaseClinical Trials	Nineth Rare DiseasePre- Clinical	Nineth Rare DiseaseOther

Do you study more than NINE Rare Diseases?

Do you study more than NINE Rare Diseases? Yes No
In order of effort, please name TENTH Rare Disease you are currently studying:
Maximum of 90 characters allowed. Currently Entered: 0 characters.

Type(s) of current activities in your TENTH Rare Disease Studies
	Animal Models	Cellular/ Molecular	Outcomes/ Comparative Effectiveness	Cellular Models	Clinical Patient Care	Clinical Trials	Pre- Clinical	Other
Tenth Rare Disease	Tenth Rare DiseaseAnimal Models	Tenth Rare DiseaseCellular/ Molecular	Tenth Rare DiseaseOutcomes/ Comparative Effectiveness	Tenth Rare DiseaseCellular Models	Tenth Rare DiseaseClinical Patient Care	Tenth Rare DiseaseClinical Trials	Tenth Rare DiseasePre- Clinical	Tenth Rare DiseaseOther

Do you study more than TEN Rare Diseases?

Do you study more than TEN Rare Diseases? Yes No
8. Do you use Animals in your research?

8. Do you use Animals in your research? Yes No
If yes, what are the Animal Models that you use?
Maximum of 150 characters allowed. Currently Entered: 0 characters.
9. Do you work with any patient Advocacy Groups?

9. Do you work with any patient Advocacy Groups? Yes No
If yes, what Patient Advocacy Group(s) and what is your role?

Maximum of 225 characters allowed. Currently Entered: 0 characters.
10. Do you currently have a therapeutic strategy under development that would benefit from a partnership with industry?

10. Do you currently have a therapeutic strategy under development that would benefit from a partnership with industry? Yes No
If yes, what Rare Disease does it target?
Maximum of 75 characters allowed. Currently Entered: 0 characters.

Survey for Penn Center for Orphan Disease Research & Therapy

Survey for Penn Center for Orphan Disease Research & Therapy

Page 1 of 2 - Questions 1-10-Information about you & your Orphan Disease Research.