MEDICAL RECORD 
CONTINUATION SHEET for either: 
NIH 2514-1, Consent to' Participate In A Clinical Research Study 
NIH 2514-2, Minor Patient’s Assent to Participate In A Clinical Research Study 
STUDY NUMBER: CONTINUATION: page JL of JL pages. 
Since this study involves a very new approach to patients with 
Gaucher disease, it will be important to maintain contact with you 
for the rest of your life, so that we can monitor your overall 
progress and monitor for any possible long term side effects. If 
the GC gene is successfully transferred into stem cells, your body 
would contain cells carrying the transferred gene for life. We 
would continue to see you in our clinic yearly for at least five 
years. In the event of your death, we would request an autopsy for 
the purpose of determining whether the gene has been transferred 
into important organs, such as the liver or spleen. 
It is important to note that participation in this study will in no 
way alter any of the standard care you may be receiving. If you are 
currently receiving enzyme replacement therapy, this should 
continue to be administered at the same dose through your primary 
physician. There will be no charge to you or your insurance carrier 
for any of the tests, procedures, or hospital stays, associated 
with this study. You will continue to be responsible for the costs 
of standard care, including enzyme replacement. You may withdraw 
from this study at any time; and we would continue to follow you as 
described above without prejudice. If you remain on the study for 
the entire year, you are then free to explore any other treatment 
options, standard or experimental. 
Potential Benefits: We do not know if there will be any direct 
benefits to you from participating in this study. The major goal 
of this study is to determine if this gene transfer approach is 
safe and feasible in Gaucher patients. However, there may be future 
benefits to you and other patients with Gaucher disease. It is 
possible that the results of this study will lead to a new form of 
therapy for Gaucher disease for you and other patients with Gaucher 
disease, or other genetic disorders. 
Potential Risks: The specific risks associated with each component 
of this protocol were described above. In any new form of therapy, 
there may be risks which are unknown or not anticipated. Since the 
vector does not replicate inside the body, we do not anticipate 
that it can alter your .sperm/ova, and therefore we would not 
anticipate that this treatment could adversely affect your 
offspring. Participation in this study will not lower the risk of 
passing Gaucher disease to your offspring. 
Woman of child-bearing potential are advised to use an effective 
method of birth control until the safety of this therapy has been 
fully established. Men are advised to use barrier-protection while 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
' [834] 
NIH-2514-1 (10-34) 
“RtetflfiMnant DNA Research, VolWirW 5059 
