Navigation Links
Shire Submits Biologics License Application (BLA) for REPLAGAL(R) With the U.S. Food and Drug Administration (FDA)
Date:12/22/2009

pproximately 20 years in men and 15 years in women, compared with the general population.[1,2] The principal causes of death are renal failure, cardiomyopathy and cerebrovascular events (e.g. stroke).[3]

Fabry disease affects an estimated 8,000 to 10,000 people worldwide.

About Gaucher disease

Gaucher disease is an autosomal recessive disorder caused by mutations in the GBA gene which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase. This enzymatic deficiency causes an accumulation of glucocerebroside, primarily in macrophages. In this lysosomal storage disorder (LSD), clinical features are reflective of the distribution of Gaucher cells in the liver, spleen, bone marrow, skeleton, and lungs. The accumulation of glucocerebrosidase in the liver and spleen leads to organomegaly. Bone involvement results in skeletal abnormalities and deformities as well as bone pain crises. Deposits in the bone marrow and splenic sequestration lead to clinically significant anemia and thrombocytopenia.

Gaucher disease is the most prevalent LSD. Gaucher disease has classically been categorized into 3 clinical types. Type 1 is the most common; it is distinguished from Type 2 and Type 3 by the lack of early neurological symptoms. Type 1 Gaucher disease is characterized by variability in signs, symptoms, severity, and progression.

Shire's velaglucerase alfa program included the largest and most comprehensive set of Phase III clinical trials conducted to date for Gaucher disease. Over 100 patients at 24 sites in 10 countries around the world have participated in the clinical studies.

Velaglucerase alfa is made using Shire's proprietary technology, in a human cell line. The enzyme produced has the exact human amino acid sequence and has a human glycosylation pattern.

    References

    1. MacDermot KD, Holmes A, Miners AH. Anderson-Fabry disease: clinical
       
'/>"/>
SOURCE Shire Plc
Copyright©2009 PR Newswire.
All rights reserved

Page: 1 2 3 4 5

Related medicine technology :

1. Shire Expands its Award-Winning ADHD Support Resource, ADHDSupport.com, by Launching an ADHD Twitter(TM) Page
2. Shire and Teva Settle Litigation Concerning Supply of ADDERALL XR Authorized Generic
3. Shire Announces Publication of Open-Label Study on Coadministration of INTUNIV(TM) (guanfacine) Extended-Release Tablets with Stimulants
4. Shire Re-Launches FOCUS, an Online, Personalized Patient Support Program for Adults with ADHD Taking Vyvanse(R) (lisdexamfetamine dimesylate) Capsules CII
5. Shire Reports Tolerability and Clinical Effects Results of Daytrana(R) (methylphenidate transdermal system) from Study in Adolescents with ADHD
6. Shire Continues to Deliver Excellent Growth From Core Products
7. Shire to Present Important Study Findings on its ADHD Treatments at a Major Psychiatric Meeting on October 29 and 30
8. Shire Presents Results of Five Lialda(R) (mesalamine) Analyses at ACG 2009 Annual Scientific Meeting
9. Shire Targets Fourth Quarter Filing of BLA for REPLAGAL(R) for Fabry Disease With U.S. FDA
10. Shire and Sandoz Settle all Pending Litigation Concerning ADDERALL XR(R)
11. Veriscrip and DAMMAD Team Up to Combat Prescription Drug Abuse in New Hampshire
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/27/2015)... , February 27, 2015 ... bahnbrechendes Überwachungssystem für kongestive Herzinsuffizienz (CHF) in ...     ... ein neuartiges Herzüberwachungssystem für Patienten mit kongestiver ... dass sie ein Finanzierungsvorhaben über 5 Mio. ...
(Date:2/27/2015)... JERUSALEM , Feb. 27, 2015  Chiasma, Inc., ... lead product for the orphan condition acromegaly, today announced ... round. Participants in the financing included new investors Rock ... chip public investment fund, as well as existing investors ... and ARCH Venture Partners. Chiasma intends ...
(Date:2/26/2015)... February 27, 2015 RnRMarketResearch.com ... market research report to its store. This report ... therapeutic pipeline. The report "Corneal Ulcers - Pipeline ... therapeutic development for Corneal Ulcers. Corneal Ulcers are ... bacterial infections, viral infections & fungal infections. The ...
Breaking Medicine Technology:Vectorious Medical Technologies schließt Finanzierungsvorhaben über 5 Mio. USD ab 2Chiasma Announces Completion of $70 Million Series E Financing 2Chiasma Announces Completion of $70 Million Series E Financing 3Corneal Ulcers Therapeutics Companies and Drugs Pipeline H2 2014 Review Research Report 2Corneal Ulcers Therapeutics Companies and Drugs Pipeline H2 2014 Review Research Report 3Corneal Ulcers Therapeutics Companies and Drugs Pipeline H2 2014 Review Research Report 4Corneal Ulcers Therapeutics Companies and Drugs Pipeline H2 2014 Review Research Report 5
... Heart, Inc. today announced that NewYork-Presbyterian Hospital/ Columbia ... U.S. center, and first in the Northeast, to ... of the DuraHeart Bridge-To-Transplant U.S. pivotal trial, a ... up to 40 centers nationwide. The trial ...
... 7 NovaBay Pharmaceuticals, Inc. (NYSE Alternext: ... novel, synthetic anti-infective products for the treatment ... bacterial, fungal, and viral infections without developing ... that NVC-422, the Company,s lead Aganocide(R) compound, ...
Cached Medicine Technology:Terumo Heart, Inc. Reports Clinical Progress in the DuraHeart(TM) U.S. Pivotal Trial as NewYork-Presbyterian Hospital/Columbia University Medical Center Implants First Patient 2Terumo Heart, Inc. Reports Clinical Progress in the DuraHeart(TM) U.S. Pivotal Trial as NewYork-Presbyterian Hospital/Columbia University Medical Center Implants First Patient 3NovaBay Pharmaceuticals Announces Lead Anti-infective Product Candidate, NVC-422, Shown to be Effective in Treating Topical Fungal Infection 2NovaBay Pharmaceuticals Announces Lead Anti-infective Product Candidate, NVC-422, Shown to be Effective in Treating Topical Fungal Infection 3NovaBay Pharmaceuticals Announces Lead Anti-infective Product Candidate, NVC-422, Shown to be Effective in Treating Topical Fungal Infection 4
(Date:3/1/2015)... Ticket Down is a reputable source for authentic tickets ... Deadheads have been anxiously anticipating this blockbuster weekend ever since ... 50 years ago back in the 60s in Palo Alto, ... death of Jerry Garcia in 1995. This beloved group ... bluegrass, folk, space rock and country to become an unforgettable ...
(Date:3/1/2015)... Indiana Fiber Network, LLC (IFN), the ... Networking, LLC (NewWays) selected IFN as primary fiber ... is well positioned to scale future backbone transport ... in rural parts of Indiana,” said Scott Reed, ... IFN Enterprise Sales Manager, IFN provides NewWays fiber ...
(Date:3/1/2015)... 2015 “ The Barrel Mill ” was ... which takes a look at the latest and coolest technology ... of NewsWatch and a technology expert, conducted the review and ... to drinks. , According to Home Distillation of Alcohol, it's ... Well, there’s a pretty cool product that’ll help anyone achieve ...
(Date:3/1/2015)... 28, 2015 CrossFit Attollo is a CrossFit ... offering a special discount to new members. Now through ... a three month contract will receive their fourth month half ... cost members $440. The three month contract is only ... then the fourth month comes in at only $55. The ...
(Date:2/28/2015)... 2015 In recent years Medical Marijuana has ... physicians as a way for them to expand their patient ... as medicine. Since the FDA still lists Cannabis as a ... to licensed physicians that can help guide them when ... Malka in Santa Cruz, CA is a leader in ...
Breaking Medicine News(10 mins):Health News:Grateful Dead Tickets at Soldier Field in Chicago: Ticket Down Slashes Grateful Dead Ticket Prices in Chicago at Soldier Field for 4th of July Weekend Celebration 2Health News:NewWays Networking Selects Indiana Fiber Network, LLC as Fiber Transport Provider 2Health News:An Oak Infusion Spiral was Featured on NewsWatch Television on January 30, 2015 2Health News:CrossFit Gym in Vancouver WA Offers Spring Special 2Health News:Reputable Cannabis Doctor In Santa Cruz and Monterey Joins the Medical Cannabis Network 2
... the lungs in bar workers according to a new study//. ... of the American Medical Association, and comes from Scotland where ... banned on March 26, 2006. ,Researchers such ... in Dundee, Scotland took this opportunity to study 77 nonsmoking ...
... of colon cancer (carcinoma) cases has been discovered by ... which could be the possible target for treatment against ... were able to rescue zebra fish from the effects ... gene, by genetically disabling (undoing) a molecule called C-Terminal ...
... "Mediterranean" diet rich in olive oil, vegetables, fruits, legumes, ... Alzheimer's disease according to U.S. researchers. ,Lead researcher ... Columbia University Medical Center in New York said, "We ... Alzheimer's disease." ,Scarmeas added that this benefit ...
... Dr Mukesh Haikerwal, said today the Government must make ... to be truly effective in meeting the needs of ... were announced today by the Prime Minister, and the ... care. ,While the AMA is generally pleased ...
... for permission with European medicine regulators to begin selling its ... move in the US last month, and comes slightly earlier ... to be one of GSK's most promising pipeline drugs as ... Code the sales of the drug could cross 2 bln ...
... under go lumpectomy feel that the outcome is a ... look, a recent survey has revealed. , ,The ... 2006 conference in San Francisco was witness to the ... of unhappy lumpectomy patients would explore the possibility of ...
Cached Medicine News:Health News:Novel Techniques to Tackle Colon Cancer 2Health News:Mental Health Package Regrettably Undervalues the Role of the GP 2
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... is a national reference laboratory and a ... development. ARUP offers an extensive test menu ... in clinical and anatomic pathology. Owned by ... include more than half of the nation's ...
Medicine Products: