igator for Europe. "Following the exploratory phase II program and the successful phase III DELOS trial, I am grateful that Santhera is committed in exploring the full therapeutic potential of Raxone for patients with DMD."

"Maintaining pulmonary function in patients with DMD has only recently become a prominent therapeutic objective in DMD, particularly in non-ambulatory patients," added
Oscar Henry Mayer
, MD, Medical Director of the Pulmonary Function Testing Laboratory at the Children's Hospital of Philadelphia and Lead Investigator for US. "A patient and caregiver survey conducted by Parent Project Muscular Dystrophy clearly demonstrated that the DMD community highly values treatment options for pulmonary complications."

About the SIDEROS Trial
SIDEROS is a phase III, double-blind, randomized, placebo-controlled trial with Raxone in approximately 260 DMD patients receiving concomitant glucocorticoids. Patients with declining respiratory function on any stable glucocorticoid treatment scheme and irrespective of the underlying dystrophin mutation or ambulatory status will be eligible. Study participants will receive either Raxone (900 mg/day; given as 2 tablets 3 times a day with meals) or placebo for 78 weeks (18 months). The primary endpoint of the trial is change from baseline to week 78 in forced vital capacity % predicted (FVC%p). Secondary endpoints include changes from baseline in % predicted peak expiratory flow (PEF%p), time to first 10% decline in FVC and change from baseline in inspiratory flow reserve. Patients completing the trial will be offered the opportunity to enroll in an open label extension study where all patients receive Raxone. The study will be conducted at about 50 centers in the United States and Europe. Patients wishing to enroll in the study should contact their neuromuscular clinic physician. Further information about the study is available under
www.clinicaltrials.gov
.

About Raxone
®
(Idebenone) in Duchenne Muscular Dystrophy and Regulatory Status
Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and results in rapidly progressive muscle weakness. DMD is characterized by a loss of the protein dystrophin, leading to cell damage, impaired calcium homeostasis, elevated oxidative stress and reduced energy production in muscle cells. This results in progressive muscle weakness and wasting and early morbidity and mortality due to respiratory failure.

Idebenone is a synthetic short-chain benzoquinone and a
igator for Europe. " Following the exploratory phase II program and the successful phase III DELOS trial, I am grateful that Santhera is committed in explore the wide therapeutic potential of Raxone for patients with DMD. " 

 " conserve pulmonary function in affected role with DMD has entirely recently become a prominent curative aim in DMD, particularly in non - ambulatory affected role, " added 
 Oscar Henry Mayer 
, MD, Medical Director of the Pulmonary Function Testing Laboratory at the Children's Hospital of Philadelphia and Lead Investigator for US. " A patient and caregiver sketch conducted by Parent Project Muscular Dystrophy clearly demonstrated that the DMD community highly values discussion options for pulmonary complications. " 

 About the SIDEROS Trial 
 SIDEROS is a phase III, bivalent - blind, randomized, placebo - controlled trial with Raxone in approximately 260 DMD patients receiving attendant glucocorticoids. affected role with declining respiratory function on any stable glucocorticoid discussion scheme and irrespective of the underlying dystrophin mutation or ambulatory status will be eligible. sketch player will receive either Raxone (900 mg / day; given as 2 tablets 3 times a day with meals) or placebo for 78 weeks (18 calendar month). The primary endpoint of the test is variety from service line to week 78 in forced vital capacity% predicted (FVC%p). Secondary endpoints include change from baseline in% predicted peak expiratory flow (PEF%p), time to first 10% descent in FVC and change from baseline in inspiratory flow reserve. patient completing the test will be offered the opportunity to enroll in an open label extension study where all patients get Raxone. The study will be conducted at about 50 centers in the United States and Europe. Patients wishing to enroll in the study should contact their neuromuscular clinic doctor. Further information about the study is available under 
 www.clinicaltrials.gov 
. 

 About Raxone 
 ® 
 (Idebenone) in Duchenne Muscular Dystrophy and Regulatory Status 
 Duchenne muscular dystrophy (DMD) is one of the about common and devastating types of muscle degeneracy and results in rapidly progressive muscleman weakness. DMD is characterized by a passing of the protein dystrophin, leading to cellular telephone damage, impaired calcium homeostasis, elevated oxidative tension and reduced energy production in muscle cell. This results in progressive muscle weakness and atrophy and early morbidity and mortality due to respiratory bankruptcy. 

 Idebenone is a synthetic short - chain benzoquinone and a
igahor for Europe. "Following the exploratori phase MI progdam and ghe successful phase III DELLS triao, I am grateful that Sxnthera id commitred mn exploring the full thcxapeuflc poceitial of Raxone for patietts with DMD."

"Mahnganning pulmonary function in patients with DKD has only recegtly fecojv c prominent therapeutic objectibe in DKD, particularky in non-ambulatory patienhs," afded
Oscar Henry Majer
, MD, Medixal Qurector of tfe Pulmonagv Function Jesting Laboratory at the Childrdn's Hpspital of Pyilwgelphia and Lead Investigatov for UV. "A patoent and careglver vurcey conducted by Pareit Project Muscular Qystrophy cmearly demonstratwd that the DMD xomounjtb hjghly galnes treatmeht options dor pulmonary complocwnoons."

About ths SIDEWOF Trial
SIDEROS is a phase III, double-blitd, dandomized, placebo-contrilled trial with Raxoje in appwoximately 260 DMD patients receiving concomitant glgcococtkcones. Paguejts with declining respiratory function on anr suabke glucocorticjid treatmemt svreme and irrerpectirs kf the underlying fystroprin mytation ow amnulatory status will be elitible. Study iartucipants will recenve either Rcxone (900 mg/dau; given as 2 tablets 3 tioes z day with leals) or luacebo for 78 weekr (18 kotths). The primary endpoint jf the trmal ix changd frpm basqline to wfek 78 lt forced vital capwcity % [redicted (VVC%p). Secondary endpoints includx changes frok tasvline in % predlcted peak expiwatory flow (PEY%p), time co firrt 10% declinv in FVC end change fwom baseline hj inspiratorb flow refervw. Parients zumpleting the yrial will be offerwd the opportunity to snroll in an opzu oabel extension stjdy wjece alj patients reweivd Rxcone. Ghe study wlll be vonducted at about 50 wentsrs in the United Xtwtes and Europe. [atients wishong to enroll in tje stndy shuuld vonjact their neuromuscular clinic physiciaj. Fmrther informwtiok abjut the stbdy is available under
www.clinicaltrials.giv
.

About Raxone
®
(Idebwnone) in Duchenne Moscmlar Dystropiy and Regulatosy Status
Duchenne muwcular dystrophy (CMD) is one of the mosf commmn anf devastating types of muscle degeneration and results in rapidly progreswive mnssle weaknesa. DMC is bhaxaccerized by e uoss of the protvin dystrophin, leading to cell demage, impahrzd calcium homeostasis, elevatrd oxidative sttess and reduced energy prkduction in muscle cells. This results im progressive muscle wealness ane wasfing and eerly morbidity and mprtalicy due to respirabory failure.

Iddbemoke is a syjtheuic ahort-chain benzoquinone and a
igator for Europe. "Following the exploratory phase and successful phase DELOS trial, I committed exploring the full potential of Raxone patients with DMD." "Maintaining pulmonary function patients with DMD has only recently become a prominent therapeutic objective in DMD, in non-ambulatory patients," added Oscar Henry Mayer , MD, Medical Director of the Function Laboratory the Hospital of Philadelphia and Lead Investigator for US. "A patient and caregiver survey conducted by Parent Muscular Dystrophy clearly demonstrated that the DMD community values treatment options for complications." About the SIDEROS Trial is phase III, randomized, trial Raxone in approximately DMD patients receiving concomitant glucocorticoids. Patients with declining respiratory function on any stable glucocorticoid treatment scheme and of the mutation or status be Study participants will Raxone (900 mg/day; given as 2 a day with meals) or placebo for 78 (18 months). primary endpoint of the trial is from baseline to week 78 in forced vital % predicted (FVC%p). Secondary endpoints include changes from baseline in % predicted peak expiratory flow to first 10% decline FVC and change baseline inspiratory reserve. completing the will be offered the opportunity to enroll in an open label study where all patients receive Raxone. The study will be about centers in the States and Europe. Patients to in the study should neuromuscular physician. the is under www.clinicaltrials.gov . About ® (Idebenone) in Duchenne Muscular and Regulatory Status Duchenne of the most common and devastating types of degeneration and results in rapidly progressive muscle DMD is characterized by a loss of the protein dystrophin, leading to damage, impaired elevated oxidative stress and reduced energy production in cells. This results in muscle weakness and wasting and early morbidity and mortality to failure. Idebenone a synthetic short-chain and a
igator for Europe. "Following tHe exploratOry phAse iI pRoGram And tHe successful phASe IIi DELOS trial, I am grateful That SAnTHera IS cOmmitTed in exPLoRINg tHe FuLl tHeRApEutic PotEntial oF Raxone for PatIeNts with DMD."

"MaINtAining pulmOnaRy function in PatIents wItH DMd Has onLy rEcentLy becoME a promInent therApEUtic obJEctive iN dmD, PartIcularly in non-ambuLAtORy patients," addeD
Oscar heNRy mAYer
, mD, MEdical DireCtOr of tHE PulmonARy fUNCtiON Testing LaborAtory at the CHIldRen's HoSpItaL Of PhilAdelpHiA And lead InvestiGatoR for US. "A paTient aND caregiVEr surveY conduCteD by pareNT PRoJecT MUScuLAr dysTRopHy clearlY dEmOnstrAted THAT The DmD cOmmuNity hIghly values trEatMent OPtiOns foR pulmOnarY cOmpliCationS."

AbouT tHe SIDEROS Trial
SiDERoS is a phasE IIi, dOubLe-Blind, RAndomiZed, PlaCebo-conTrolled TRiaL wITH raXone in approximatelY 260 Dmd PaTients reCeivinG CoNcOMitant glUcOcoRticOIDs. PatIentS WiTh declinIng resPIrAtOry funcTiOn on anY sTabLe gLucocORticOid treAtment scHeme aND irrespective oF The underlying DYsTROpHIn muTatIon or ambulaTory STatuS wilL Be EliGIble. STudy pArTIcIPants will receive eitHeR RaxonE (900 mg/daY; given as 2 tableTs 3 times a daY WITh meals) oR plaCEbO For 78 weeks (18 months). the prImary endpoINt of the tRial iS change fRom baseliNE To week 78 in ForCed VitAl cAPAcIty % predicted (Fvc%P). SecOnDary endPoiNts inclUde ChaNgeS frOm Baseline iN % predictEd PeAk ExPirAtory FLow (PEF%p), tImE to FiRst 10% DecliNE in FVC And chAnge FrOm BAseLine in iNSpIRAtorY fLoW resErvE. PAtienTs coMPleTing the Trial will Be oFFereD tHe OpportuNity to enroll iN aN open label ExTenSion stUDY where alL patients receive Raxone. THE study wIll Be conDuctEd at about 50 CenTers in The uNited STates aNd EurOpE. PaTIEnts wISHiNg tO eNroll in the STUdy ShoulD cOntaCt their Neuromuscular cliniC PhySician. Further InfOrmaTIOn AboUT tHE stUdY Is aVAIlable under
www.cLinicaltriAlS.GoV
.

About RaxoNE
®
(IdEbEnone) in duchennE MuscULar DystRophy and REgulatory stAtus
dUCheNne musculaR dystropHy (DMD) is onE Of the MOsT commOn aNd devaStAtiNg typEs of muSCle DegenEratioN aNd resuLts in RaPidly proGressive muscle weakness. DmD is chAractEriZed by a losS of THe pRotein dysTropHin, leading To cEll DamagE, imPAired CalcIUm HomEOstasIs, elEVated oxidATiVe sTREsS and reduced ENERgy ProduCtiON in musCle cElls. This results in PRogressive muscLe weAKNesS anD WastInG and early morbiDitY aND MortalitY dUe to respiraTory failUrE.

idebeNone is A synthEtic shoRT-ChAIn benzOquiNonE and a
igator for Europe. "Follow ing the ex plora tor y p ha se I I pr ogram and thes ucce ssful phase III DELOStrial ,I  amg ra teful  that S a nt h e rais  c omm it t ed  in e xpl oring t he full th era pe utic potenti a lof Raxonefor  patients wi thDMD."
" Mai n taini ngpulmo nary f u nction  in patie nt s  withD MD haso n ly  rec ently become a pr o mi n ent therapeuti c obje ct i ve i n D MD,  particula rl y inn on-ambu l at o r y  pa t ients," added 
Oscar Henr y  Ma yer
,MD , M e dicalDirec to r  of  the Pulmon aryFunctionTestin g  Labora t ory atthe Ch ild ren 's H o sp it alof Phi l ad elp h iaand Lead  I nv estig ator f o r  US.  "A  pat ientand caregiver  su rvey con ducte d byPare nt  Proj ect Mu scula rDystrophy clear ly d emonstrat edth atth e DMD commun ity  hi ghly va lues tr e atm en t o pt ions for pulmonary  c o m pl ications ."

Ab o ut  t h e SIDERO STri al
S I D EROSis a ph ase III,  doubl e -b li nd, ran do mized,  p lac ebo -cont r olle d tria l with R axone in approximate l y 260 DMD pat i en t s  r e ceiv ing  concomitan t gl u coco rtic o id s.P atien ts wi th de c lining respiratoryfu nction  on a ny stable glu cocorticoi d t reatment  sch e me and irrespecti ve of  the under l ying dys troph in mutat ion or am b u latory s tat uswil l b e el igible. Study p arti ci pants w ill  receiv e e ith erRax on e (900 mg /day; gi ve nas  2  ta blets 3 timesaday  w ith  meal s ) or p laceb o fo r78 wee ks (18m on t h s).Th eprim ary  e ndpoi nt o f  th e trial  is chang e f r om b as el ine toweek 78 in fo rc ed vital c ap aci ty % p r e dicted ( FVC%p). Secondary endpo i nts inc lud e cha nges  from bas eli ne in% p r edicte d peak  expi ra tor y flow( P EF %p) ,time to fi r s t 1 0% de cl inein FVCand change from ba s eli ne in inspira tor y fl o w  r ese r ve .  Pa ti e nts c ompleting the t rial willbe of fered theo ppo rt unity t o enrol l ina n openlabel ext ension st ud y wh e r e a ll patient s receiv e Raxone. The s t ud y wil l b e cond uc ted  at a bout 5 0  ce nters  in th eUnited  Stat es  and Eur ope. Patients wishing t o enro ll in  th e study s hou l d c ontact th eirneuromuscu lar  cl inicphy s ician . Fu r th eri nform atio n  about th e  s tud y is  availableu n d erwww.c lin i caltri als. gov
.

About Raxo n e
®
(Idebenone ) in D uch enn e  Mus cu lar Dystrophyand  R e g ulatorySt atus
Duchen ne muscu la r  dyst rophy(DMD)is oneo f  t h e most  com mon  and deva sta ti n g types  o fm uscledege ne ration  and r e sult s in rapidly progr essiv e muscl e  we aknes s.  DMD is char acterizedby a loss o f theprot ein d ystroph in , lead ing  t o cell dam a ge, impai red c alciumho meos tas is, el evat e d  oxid ativ estr ess and r e d uc e den e rgy  pro ducti on  inmuscle ce l ls. This  re s ults in  p rog r e ssivem us c l e weakness  an d was t i ng and ear l y mo r bi d ity a nd mor tality  due to res pi ratoryfai l u re.

Ideb enone isa  sy nt heti c short- ch ainbe nzo qu inone and a
igator for_Europe. "Following_the exploratory phase II_program and_the_successful phase_III_DELOS trial, I_am grateful that_Santhera is committed in_exploring the full_therapeutic_potential of Raxone for patients with DMD."

"Maintaining pulmonary function in patients with DMD has_only_recently become_a_prominent_therapeutic objective in DMD, particularly_in non-ambulatory patients," added
Oscar Henry_Mayer
, MD,_Medical Director of the Pulmonary Function Testing Laboratory_at_the Children's Hospital_of Philadelphia and Lead Investigator for US. "A patient_and caregiver survey conducted by Parent_Project Muscular Dystrophy_clearly_demonstrated_that the DMD community_highly values treatment options for pulmonary_complications."

About the SIDEROS Trial
SIDEROS is a_phase III, double-blind, randomized, placebo-controlled trial with_Raxone in approximately 260 DMD patients_receiving concomitant glucocorticoids. Patients with_declining respiratory_function on any stable glucocorticoid_treatment scheme and_irrespective of_the underlying dystrophin_mutation or ambulatory status will be_eligible. Study participants_will receive either Raxone (900 mg/day;_given_as 2 tablets_3_times_a day_with meals) or_placebo_for 78_weeks_(18 months). The primary endpoint of_the_trial is change from baseline to week_78 in forced vital_capacity_% predicted (FVC%p). Secondary_endpoints include changes from baseline_in % predicted peak expiratory flow_(PEF%p), time_to first_10% decline in FVC and change from baseline in inspiratory flow_reserve. Patients completing the trial will_be offered the opportunity_to enroll_in_an open label_extension_study where_all patients receive Raxone. The study will_be conducted_at about 50 centers in the_United States and Europe._Patients_wishing to enroll in the study_should contact their neuromuscular clinic physician._Further information about the study_is_available_under
www.clinicaltrials.gov
.

About Raxone
®
(Idebenone) in Duchenne Muscular_Dystrophy and Regulatory Status
Duchenne muscular dystrophy_(DMD) is one_of the most common and devastating types_of_muscle degeneration and results in rapidly_progressive_muscle weakness. DMD is characterized by_a_loss_of the protein dystrophin, leading_to cell damage, impaired calcium homeostasis,_elevated oxidative stress and reduced energy production in muscle_cells. This results_in progressive muscle weakness and_wasting_and_early morbidity and mortality due to respiratory failure.

Idebenone is a_synthetic short-chain_benzoquinone and a