Main > > >GENETIC DISEASES.

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Adenosine Deaminase Severe Combined ImmunoDeficiency (ADA-SCID). Children.
EU Approval Date: 2016. 05.30.
(*) Company : Orchard Therapeutics.
www.orchard-tx.com.
TradeMark: Strimvelis.
UpDate: 2018. 04.13.




Amyloidosis TransThyRetin PolyNeuropa-
try. TREAT.: Inotersen Na Inj. 284 mg/
1.5 ml.
CA Approval Date : 2018. 10.04.
EU Approval Date : 2018. 07.11.
USA Approval Date: 2018. 10.05.
(*) Company; Patents; TradeMark & Web-
Sites Available on Request.
UpDate: 2018. 10.09.




Amyloidosis TransThyRetin PolyNeuropa-
thy. TREAT.: Tafamidis Capsules. (U>P)
EU Approval Year: 2011.
JP Approval Year: 2013.
Also Approved: AR, BR, IL, KR, MX & RU.
(*) Company; Patents; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 08.12.




AngioEdema. Preventative Treat.: C1 Es-
terase Inhibitor SubCutaneous (SC) In-
jection.
USA Approval Date: 2017. 06.22.
USA Launch Date : 2017. 07.25.
(*) Company; Patent; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 08.30.




AngioEdema. Preventative Treat.: Lanade luMAb SubCutaneous Injection.
USA Approval Date: 2018. 08.23.
(*) Company; Patents; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 08.29.




AngioEdema. Treat.: C1 Esterase Inhi-
bitor SubCutaneous (SC) Injection. (N)
Conestat Alfa.
EU Approval Date : 2014. 02.24.
IL Approval Date : ?
USA Approval Date: 2014. 07.16.
(*) Company; Patent, TradeMark & Web-
Site Available on Request.
UpDate: 2018. 08.30.




AngioEdema. Treat.: C1 Esterase Inhi-bitor SubCutaneous (SC) Injection. (U)
Approved in: AR, AU, CA, DE, IL, JP, KR,
& USA.
(*) Company; Patent; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 08.30.




Duchenne Muscular Dystrophy. TREAT.:
Eteplirsen Inj.
USA Approval Date: 2016. 09.19.
(*) Company; Patents; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 07.30.




Fabry Disease. TREAT.: Migalastat.HCl.
AU Approval Date : 2017. 08.15.
CA Approval Date : 2017. 09.14.
ES Launch Date : 2018. 01.18.
EU Approval Date : ?
FR Launch Date : 2017. 05.02.
JP Launch Date : 2018. 05.30.
USA Approval Date: 2018. 08.10.
(*) Company & TradeMark Available on Request.
UpDate: 2018. 08.11.




Fragile X Syndrome. DIAG.
USA Approval Date: 2020. 02.24.
(*) Company; Patents; TradeMark & Web-
Site Available on Request.
UpDate: 2020. 04.30.




Gaucher Disease Type I. TREAT.:
EliGlustat Capsules.
CA Approval Date : 2017. 05.01.
USA Approval Date: 2014. 08.19.
(*) Company; Patents; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 04.24.




Gaucher Disease Type I. TREAT.:
MiGlustat Capsules. Generic.
USA Approval Date: 2018. 04.23.
(*) Company & Patents Available on
Request.
UpDate: 2018. 04.24.




MucoPolySaccharidosis VII. TREAT.:
Vestronidase Alfa Injec
tion for IV Use.
(MPS VII, Sly Syndrome)
USA Approval Date: 2017. 11.15.
(*) Company; TradeMark & Web-Site Available on Request.
UpDate: 2018. 03.14.




Ophthalmo.>Treat.: Voretigene Neparvovec rzyl. (U)
(Inherited Retinal Disease due to Mutations in Both Copies of the RPE65 gene)
USA Approval Date: 2017. 12.19.
(*) Companies; Patent; TradeMark &
Web-Site Available on Request.
UpDate: 2018. 07.03.




PhenylKetonUria. TREAT.: PegValiase-pqpz
Injection.
EU Approval Date: 2019. 05.06.
USA Approval Date: 2018. 05.24.
(*) Company; Patents; TradeMark & Web-
Site Available on Request.
UpDate: 2019. 05.07.




PhenylKetonUria. TREAT.: SaproPterin.
2HCl. Oral. (C)
(*) Company; Patent; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 05.26.




PhenylKetonUria. TREAT.: SaproPterin.
2HCl. Oral. (T)
Company received Exportation License in 2013.
(*) Company; Patent & Web-Site Avai-
lable on Request.
UpDate: 2018. 05.26.




PhenylKetonUria. TREAT.: SaproPterin.
2HCl. Oral. (U)
EU Approval Date : 2008. 12.09.
JP Approval Date : 2008. 07.16.
USA Approval Date: 2007. 12.13.
(*) Company; Patents; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 05.26.




Primary Hyperoxaluria (PH) Type 1.
Treat.: Lumasiran for Inj.
Endocrinology; Urology.
EU CE Mark Date : 2020. 11.19.
USA Approval Date: 2020. 11.24.
(*) Company; Patent; TradeMark & Web-
Site Available on Request.
UpDate: 2021. 03.19.




Spinal Muscular Atrophy. Treat.: Nusi-
nersen Inj.
CA Approval Date : 2017. 07.04.
EU Approval Date : 2017. 06.01.
USA Approval Date: 2016. 12.23.
(*) Company; Patents; TradeMark & Web-
Site Available on Request.
UpDate: 2018. 12.10.




Tyrosinaemia Type 1. TREAT.: Nitisinone. Capsule & Oral. (S)
Suspension Formulation.
Capsule>CA Approval Date: 2017. 01.04.
Capsule>SA Approval Date: 2017. 05.17.
Capsule>USA Approval Date: 2016. 04.26. (Twice Daily)
Capsule>USA Approval Date: 2017. 09.05. (Once Daily)
Oral Suspension Formulation>CA Approval Date: 2017. 11.27.
(*) Company; TradeMark & Web-Site Avai-
lable on Request.
UpDate: 2017. 11.28.




Tyrosinaemia Type 1. TREAT.: Nitisinone. Tablet. (U)
USA Approval Date: 2017. 08.01.
(*) Company; Patent APP.; TradeMark &
Web-Site Available on Request.
UpDate: 2017. 11.28.



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