,,iv  #aai? ,,congress  #a/ ,session    
,h4 ,,res4 #bji  ,,9 ,,! ,,h\se ,,(     
,,repres5tatives  ,m>* #aj1 #bjbe,mr4   
,tonko 7= himself1 ,mr4 ,bilirakis1 ,ms4
 ,matsui1 ,mr4 ,norman1 ,mrs4 ,trahan1  
,mr4 ,wilson ( ,s\? ,c>ol9a1 & ,mr4     
,au*9closs7 submitt$ ! foll[+           
resolution2 :i* 0 ref]r$ to ! ,committee
 on ,en]gy & ,comm]ce  ,,resolution     
,express+ support = ! designation (     
,april #e1 #bjbe1 z ,b>? ,syndrome      
,aw>5ess ,day4  ,:]eas ,b>? syndrome is 
a r>e1 life-?reat5+1 g5etic disord] :i* 
prim>ily affects males2 ,:]eas ,b>?     
syndrome is caus$ by a mutation 9 !     
tafazz9 g5e 7,,tafazz91 also call$      
,g#d4e71 result+ 9 an inborn error (    
phospholipid metabolism1 affect+ _m     
sy/ems ( ! body2 ,:]eas ,b>? syndrome is
 a multisy/em disord] ) *>act]i/ics     
includ+ abnormalities ( he>t & skeletal 
muscle1 l[ levels ( c]ta9 :ite blood    
cells call$ neutrophils t help to fight 
bact]ial infections1 & delays 9 gr[?2   
,:]eas ) probably few] ?an #aj new ,b>? 

infants id5tifi$ ea* ye> 9 ! ,unit$     
,/ates1 ! incid5ce ( ,b>? syndrome is   
likely only #a 9 e #cjj1jjj to #djj1jjj 
bir?s & c\ld be z few z #a4e 9 #a       
million2 ,:]eas globally !re are        
approximately #cjj individuals diagnos$ 
) ,b>? syndrome1 &1 9 ! ,unit$ ,/ates1  
!re are few] ?an #aej individuals       
diagnos$ ) ,b>? syndrome2 ,:]eas ,b>?   
syndrome c be fatal 9 *ildhood due to   
he>t failure or uncontrollable          
infection1 ) approximately #ej p]c5t (  
dea?s due to ,b>? syndrome occurr+ )9 ! 
fir/ ye> ( life & #he p]c5t be=e ! fif? 
ye> ( life2 ,:]eas individuals ) ,b>?   
syndrome :o survive to adul?ood (t5 h a 
sev]ely limit$ life expectancy2 ,:]eas  
individuals ) ,b>? syndrome1 l _m o!r   
r>e disord]s1 exp]i5ce *all5ges ) obta9+
 a diagnosis1 limit$ treatm5t options1 &
 difficulty f9d+ & access+ treatm5t     
c5t]s & physicians ) exp]tise 9 ,b>?    
syndrome2 ,:]eas1 because ! disord]     
affects multiple sy/ems ( ! body1 a     
pati5t ) ,b>? syndrome (t5 requires     

access to c>e f exp]ts across a range ( 
m$ical specialties2 ,:]eas ! ,b>?       
,syndrome ,cl9ic at ,k5n$y ,krieg]      
,in/itute is ! only int]discipl9>y cl9ic
 d$icat$ to ! diagnosis & treatm5t (    
,b>? syndrome 9 ! ,unit$ ,/ates2 ,:]eas1
 to date1 !re is no treatm5t = ,b>?     
syndrome t is approv$ by ! ,food & ,drug
 ,adm9i/ration 7,,fda72 ,:]eas !re is a 
critical ne$ = rese>* & developm5t to   
advance treatm5ts = ,b>? syndrome2      
,:]eas1 z a result ( ! ,orphan ,drug    
,act1 !re h be5 important advances 9    
rese>* on & treatm5t = r>e diseases1    
includ+ developm5t ef=ts 9 ,b>?         
syndrome2 ,:]eas ! ,,fda e/abli%$ !     
,accel]at$ ,approval ,pa?way 9 #aiib &  
,congress codifi$ ! pa?way 9 #bjab2     
,:]eas ! ,accel]at$ ,approval ,pa?way is
 an important pa?way = r>e & ultr>>e    
diseases z x all[s = e>li] approval (   
drugs t treat s]i\s conditions1 & fill  
an unmet m$ical ne$ '''2 ,:]eas         
,congress & ! ,,fda h affirm$ !         
importance ( incorporat+ ! pati5t       

p]spective ?r\gh\t ! drug review process
 ?r\gh ! ,,fda's ,pati5t-,focus$ ,drug  
,developm5t program2 ,:]eas ! ,b>?      
,syndrome ,f\ndation1 a nonpr(it        
organization e/abli%$ 9 #bjjj to        
accel]ate progress ?r\gh collaboration  
betwe5 families & sci5ti/s1 is d$icat$  
to educat+1 advanc+ treatm5ts1 & f9d+ a 
cure = ,b>? syndrome2 ,:]eas ! ,b>?     
,syndrome ,f\ndation sponsors ,b>?      
,syndrome ,aw>5ess ,day 9 ! ,unit$      
,/ates to increase public aw>5ess &     
g5]ate additional support = ,b>?        
syndrome2 & ,:]eas ,b>? ,syndrome       
,aw>5ess ,day is expect$ to be obs]v$ 9 
! ,unit$ ,/ates = ye>s to come1 provid+ 
hope & in=mation = pati5ts1 c>egiv]s1 & 
families ar\nd ! c\ntry3 ,n[1 !re=e1 be 
x ,t ! ,h\se ( ,repres5tatives-- 7#a7   
supports ! designation ( ,b>? ,syndrome 
,aw>5ess ,day2 &  7#b7 recognizes !     
importance (1 ) respect to ,b>?         
syndrome-- 7,a7 improv+ aw>5ess2  7,b7  
enc\rag+ accurate & e>ly diagnosis2     
7,c7 advanc+ rese>*2  7,d7 develop+ new 

treatm5ts1 diagno/ics1 & cures2 &  7,e7 
id5tify+ regulatory pa?ways = drug      
developm5t ( ultr>>e diseases l ,b>?    
syndrome4