everyone. Brad you, good and Thank morning,
Moving to Slide Pompe for our on therapy next-generation start on we'll with XX, novel R&D updates disease. our AT-GAA,
First, therapeutic to fatal of it's crucial. is continues a choices Pompe pose and always is having important a disease neuromuscular to by Pompe for severe recognize people disorders. health of most the that challenges and the lysosomal options and disease affected and one prevalent range
and In key tragedies of Pompe history human followed often individuals. measures of patients the studies treatment addition that that seen to of is highlight individual long-term initial for disease by natural on decline the many multiple publications we've benefit continued
been means in ever studied X clinical lay PROPEL onset unmet ERT high to As trial. population All date only Phase for our Pompe studies a naïve been the a have this in has other to participants setting controlled disease in treatment. experienced controlled reminder, sustaining
Moving a summary the on we biomarker endpoints Phase Slide XX, to of PROPEL primary secondary study. present X from our key, and
reminder, and safety and against experienced treatment AT-GAA efficacy approved of double adult alglucosidase the ERT naïve set alfa. study patients therapy a blind, As in the randomized, was in
into Pompe alfa the key of ERT the patients. We can across overall illustrates these this in in of domains outcomes populations. reported motor for see Here alglucosidase potential majority domains consistency all experience endpoints AT_GAA manifestation function, the endpoints these slide, patient favor of both muscle biomarkers, impact and effects and function, across AT_GAA the of the strength, you disease pulmonary believe grouping of
with As our significance alfa statistical XX superiority. groups primary walk difference alglucosidase six-minute patients reminder the distance did reach of of the on those quite population between which for AT-GAA meters, the outperformed endpoint on on overall in not
the treatment leading pulmonary the cause important of population, with this statistically capacity percent P really difference However, endpoint overall was superiority the And FVC, for mortality in alglucosidase This of alfa, finding and Pompe. AT-GAA vital loss X.XXX. or for a versus a of value is key showed is X% secondary as on clinically difference progressive main the and of first the nominally everyone product a function alglucosidase significant approval predicted that versus meaningful which initially not upon endpoint alfa for was placebo, the study [indiscernible] PROPEL of remind superiority the approved. that the approved an therapy, tested second generally versus We and also we of Phase improve demonstration only be pleased the results prestigious in published shortly to very medical to that requires X noninferiority We're therapy. to a expect journal. announce PROPEL peer-reviewed in
updates on have Moving key the AT-GAA we to Slide program. highlighted XX, across
the XXXX, regulatory for BLA have and announced. NDA XX, May BLA. two and PDUFA for for the on migalastat, First, for date XXXX, FDA has we set progress, accepted NDA; the components U.S. These July previously cypaglucosidase for alpha the for review as the XX, the action
to As John filings be we mentioned, expect these together. reviewed
opinion positive announced granted early also a positive announced, that we June, Medicines opinion living as European the for are scheme In positive medical and for alglucosidase really those We medicine are the excited grown, to the AT-GAA marketing and physicians have need patients leading access MEA Since pleased unmet be the are momentum access that pleased MHRA. to access eligible opinion to through UK's late-onset Pompe received to now centers. the provide AT-GAA to the We're in has scientific John UK. significant permits the share, across community recognizes AT-GAA with the and by in the who least enthusiasm the eligible the years two June, in who mechanism scientific has Pompe to UK. the Pompe to that at alpha to submitted having and able we by interest to Agency. for to multiple been high AT-GAA under authorization also faced have adults switch requested was for prior to This access and see with
treated of to pleased age, onset younger point, to continue Pompe At Pompe possible. the with XX expand complete look we're For access late-onset patients ongoing both enroll received, individuals. expand XXX and programs requests Pompe to into with to years living patients those the and than more children response are expanded to worldwide many AT-GAA. that we up disease in that late-onset to this onset for access infantile study finally, And report for we've the soon as being expanded community, as disease with in open-label living disease infantile continue with multiple to our
for innovative advanced Moving of is for rare on manufacturability see Caritas. as now launch children transform and around to highlight current briefly Slide significant through the everyone, and the the overcome XX, mission protein therapies safety, diseases Caritas of gene living remind technologies. rare with to To Caritas our for and John planned engineering portfolio of industry-leading to challenges and genetic immunogenicity. noted, vector gene We we opportunities therapy lives adults durability, diseases
to We the realize therapy. programs. promise the have are Caritas portfolio developing and preclinical and technologies and both address of tools clinical The includes and gene these challenges
as We've two and INDs RET, of the rare dystrophies, diseases, the XX In a of rare expect nearly This to diseases approximately University diseases, collaboration myotonic CLNX other CLNX, new rights Fabry dozen with the the rare as including lysosomal disclosed the to and muscular Pompe. dystrophy for Batten Pennsylvania. that include see include XX in we including addition programs previously Angelman, three and diseases our in DMD. majority larger genetic to clinic which well these also multiple next global the includes months in addition to over
University with attractive Pennsylvania cost we with an will partnership. as and risk as As continue part this of Amicus sharing well through transaction, work jointly of to the
manufacturing planned with clinical Thermo the expansion foot For our manufacturing, continued with on state-of-the-art capabilities we designed, front. Fisher together with ready-to-build, fully relationship CDMO and facility have XX,XXX strong square a commercial
targeting. stability optimized internal disease potency a and Fabry of an the is Caritas programs. greater to. approach programs discovery organization protein being Slide programs, of research those rights co-development staffed and fifty-fifty Caritas will by also global highlights share this leading The with these fully differentiated These the split. Pompe ensure Pompe and a experienced of The Amicus and derisks relationship therapy in which and for entrepreneurs on Fabry experts team the and partnership continued funding engineering gene Amicus cross-correction through with provides All gene a problem solvers. the therapy leadership and and profit passionate working between programs partnership and will led development utilize XX profit-sharing and of
to expect to of next second We around first the the the comments on two build John's on Last Fabry in IND therapy half like earlier children reiterate sentiment the long-term to of study. be during period of program, would I the filed loss and the Batten our gene CLNX follow-up year. the
As both participants investigator that from disease-related to away mentioned, unrelated the drug. the John passed deemed study complication
to continue available We information. all assess
clinical study ATGCXXXX X/X As of the a enrolled reminder, XX of received who genomes. XX a at participants vector a our single Phase injection dose intrathecal to all X.X
The years infancy during disease adolescence. Batten indicates five beyond received their in are CLNX most early diagnosed over first gene literature ago. and study with not early childhood survive transport that or the individuals and children Medical do childhood
broadly. While outlook. and the results, Officer, nature Chief Financial benefits review would call our to the financial see news, and this Quimi, to our potential now gene like extremely disorder turn the to Daphne? over therapy devastating of continue we recognize that, to Daphne I guidance With of
