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Fabry sufferers, others with rare conditions mark international ...  -  Feb 29, 2008
ChronicleHerald.ca,Members of the Fabry Disease Society of Nova Scotia are pleased the province has proclaimed the awareness day. Only about 250 Canadians have the rare

VIDEO from Medialink and Shire plc: Rare Diseases Must Be Top of ...  -  Feb 29, 2008
FOXBusinessFabry Disease (also known as Anderson-Fabry Disease) is a lysosomal storage disease with an estimated birth incidence of 1 in 117000.

Amicus Therapeutics Presents Positive Data from Phase 2 Clinical ...  -  Mar 13, 2008
PharmaLive.com (press release),Amicus has completed Phase 2 clinical trials of Amigal(TM) for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for

BUYINS.NET: TLB, NGD, CTIC, FOLD, OPXA, PALM Have Been On BUYINS ...  -  Mar 14, 2008
Trading Markets (press release),The company's products include Amigal, which has completed Phase II clinical trial for the treatment of Fabry disease; Plicera, a Phase II clinical trial

Food for Thought  -  Mar 4, 2008
Morris Daily Herald,...“You need to start eating healthy,� the doctor said minutes before I was to undergo my most recent IV treatment for Fabry disease.

Alberta teen pleads for $500000 drug therapy  -  Feb 29, 2008
CBC Ottawa,Koning himself suffers from Fabry disease, a disorder that interferes with the body's ability to break down fatty substances. "It's to make our politicians

SWLA's Children's Miracle Network Champions  -  Mar 18, 2008
KPLC-TV,Nathan and Bradley Rowell were diagnosed three years ago with a rare genetic disorder - in fact, they are the only known twins to have Fabry's disease.

Enzyme replacement improves cardiomyopathy in Anderson-Fabry disease  -  Feb 18, 2008
Medicexchange,Enzyme replacement with agalsidase alfa results in regression of hypertrophic cardiomyopathy in patients with Anderson-Fabry disease, an X-linked

Samaritan to Launch REPLAGAL® for Fabry Disease in Greece and Cyprus  -  Feb 5, 2008
Free press releases (press release),REPLAGAL(r) is a long-term enzyme replacement therapy used to treat patients with a confirmed diagnosis of Fabry Disease.

Amicus Therapeutics Announces Fourth Quarter and Full Year 2007 ...  -  Jan 29, 2008
CNNMoney.comIn December 2007, Amicus announced positive results from its Phase 2 clinical trials of Amigal(TM) for Fabry disease. 26 patients completed the study,

Genzyme Corp swings into the black for fourth quarter  -  Feb 14, 2008
Pharma Times (subscription),Revenues rose 21% to $1.04 billion, driven by sales of Gaucher disease treatment Cerezyme (imiglucerase), up 15% to $301 million and Fabry disease drug

Drug maker Shire promises 680 jobs  -  Feb 13, 2008
Boston Globe,Shire's Human Genetic Therapies unit, which develops drugs to address rare medical disorders such as Fabry disease, is based in Cambridge,

Amicus Therapeutics to Present at the BIO CEO & Investor ...  -  Feb 7, 2008
CNNMoney.comAmicus has completed Phase 2 clinical trials of Amigal for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the

Genzyme Meets Views, Sees Strong Revenue  -  Feb 13, 2008
TheStreet.comFabry disease treatment Fabrazyme and Pompe disease treatment Myozyme generated sales of $114 million and $62 million, respectively.

Genzyme Delivers Strong Fourth Quarter to Conclude Outstanding Year  -  Feb 14, 2008
Lifescience-online,The European Commission has granted full marketing authorization for Fabrazyme, making the product the only Fabry disease treatment to earn this designation

Biotech Company Specializes In Treatments For Rare Disorders  -  Jan 23, 2008
CNNMoney.comThe drug helps treat Fabry's disease, estimated to affect 5000 to 7000 people worldwide. The firm's emerging segments, including its renal franchise,

Genzyme Turns To Profit In Q4 On Strong Drug Sales; Guides Q1 ...  -  Feb 13, 2008
RTT News,Sales of the company's Fabry disease treatment Fabrazyme increased 18% to $114 million from $97 million in the same quarter last year. For Fiscal year 2007,

British drugmaker Shire to expand in Massachusetts, add 680 jobs  -  Feb 14, 2008
PR-Inside.com (Pressemitteilung),...in developing treatments for genetic and rare disease. The unit's key products are Elaprase, to treat Hunter syndrome, and Replagal, for Fabry disease.

Little Daisy's life was saved thanks to a  -  Jan 24, 2008
Checkbiotech.org (press release),Fabry disease - a disorder which affects blood vessels including in the brain, kidneys and heart. The centre anticipates five to 10 new cases each year.

Cayuga Fund Up 6% in Volatile Year  -  Jan 24, 2008
Black Enterprise,A compound developed to treat the Fabry disease received mixed results in clinical trials last month, sending the stock spiraling downward and dealing a

Amicus sees positive Phase II data for Fabry disease candidate  -  Dec 19, 2007
ReutersO: Quote, Profile, Research) said it had positive results from Phase II clinical trials of Amigal for Fabry disease. Based on the results, Amicus said it

Fabry's drug outlook sinks Amicus shares  -  Dec 20, 2007
BusinessWeekLate Wednesday, Amicus said drug candidate Amigal met safety and tolerability goals in a midstage study for Fabry's disease, a rare hereditary disorder that

Amicus Therapeutics Announces Positive Results From Phase 2 ...  -  Dec 19, 2007
CNNMoney.comThe four open-label, multi-national Phase 2 trials of Amigal enrolled 18 men and 9 women with Fabry disease between the ages of 17 and 65.

Amicus Therapeutics Reports Positive Phase 2 Data Of Amigal For ...  -  Dec 19, 2007
Trading Markets (press release),Also, the data demonstrated that Amigal has a meaningful effect on a range of genetic mutations in Fabry disease and thus has the potential to treat a

Biotech Notebook: Genitope, Amicus, Introgen  -  Dec 21, 2007
TheStreet.comAmicus Therapeutics (FOLD - Cramer's Take - Stockpickr) presented some phase II data on its Fabry disease drug Amigal on Wednesday.

Amicus Experimental Drug Meets Goals  -  Dec 19, 2007
Houston Chronicle,Fabry disease is caused by genetic mutations in the GLA gene, which result in deficient activity of an enzyme called a-GAL. Deficient a-GAL can ultimately

FOLD: Plunges to 52-Week Low Despite Positive Ph II Data for ...  -  Dec 20, 2007
Therapeutics Daily (subscription) (press release),Fabry disease is caused by the lack of or faulty enzyme needed to metabolize lipids, fat-like substances that include oils, waxes, and fatty acids.

Amicus reports positive results for Amigal  -  Dec 21, 2007
Trading Markets (press release),PowerRating -- Amicus Therapeutics has presented positive results from its recently completed Phase II clinical trials of Amigal for Fabry disease.

Genzyme "outperform"  -  Dec 20, 2007
newratings.com,...the analysts mention that the Phase II data for Amicus’ Amigal drug, which competes with Genzyme’s Fabrazyme for the treatment of Fabry's disease,

$40.5m in incentives pledged to keep drug firm's plans on track  -  Dec 21, 2007
Boston Globe,Shire makes several drugs, including Elaprase for Hunter syndrome and Replagal for Fabry disease. As part of the expansion project, the company would move

Amicus Therapeutics to Host R&D Day  -  27 Nov 2007
CNNMoney.comThe presentation will include a summary of the results of the Phase 2 clinical trials performed for Amigal(TM) (migalastat hydrochloride) for Fabry disease

amicus therapeutics and shire plc enter into $440 million ex-us ...  -  Nov 8, 2007
CNNMoney.com...trials for the treatment of fabry disease, plicera(tm) (isofagomine tartrate) currently in phase 2 clinical trials for the treatment of gaucher disease,

shire expands its human genetic therapies pipeline through in ...  -  Nov 8, 2007
CNNMoney.com...which includes replagal(tm) for fabry disease, elaprase(tm) for hunter syndrome and ga-gcb in phase 3 development for gaucher disease. in addition,

amicus, shire team on rare gene r&d  -  Nov 8, 2007
TheStreet.com...(shire also owns replagal to treat the genetic disorder fabry disease.) the diseases belong to a class of lysosomal storage disorders, which are caused by a

shire signs rare disease drug licensing deal with amicus  -  Nov 9, 2007
Checkbiotech.org (press release),...three investigational compounds that treat rare genetic disorders. shire has licensed the ex-us rights to amicus’ amigal (migalastat) for fabry disease,

shire pads genetic disease therapeutic pipeline through $440m ...  -  Nov 8, 2007
Genetic Engineering News (press release),...million upfront for three compounds in markets outside of the united states: phase ii amigal™ for fabry disease, phase ii plicera™ for gaucher disease,

lsd deal brings amicus $50m now, $390m later  -  Nov 8, 2007
BioWorld Online,...shire gets ex-us development and commercialization rights to the phase ii fabry disease drug amigal (migalastat hydrochloride), the phase ii gaucher

thursday's health winners & losers  -  Nov 8, 2007
TheStreet.com...goucher's disease and fabry disease. shares rose 67 cents, or 4.2%, to $16.75. elsewhere, earnings ruled (and sometimes ruined) the day. king pharma (kg

shire partners with amicus on development  -  Nov 8, 2007
Conde Nast Portfolio,...mass., said the compounds will be used to develop therapies for lysosomal storage disorders, including fabry disease, gaucher disease and pompe disease.

update 1-amicus therapeutics signs licensing deal with shire  -  Nov 9, 2007
Reuters...a company that developed a drug called replagal to treat the genetic disorder fabry disease. shire also sells elaprase, a drug for hunter syndrome.

shire licenses drugs for rare diseases mass high tech: the journal ...  -  Nov 8, 2007
Bizjournals.com,...in markets outside of the united states. new jersye's amicus licensed amigal for fabry disease (phase 2), plicera for gaucher disease (phase 2),

amicus, shire to develop and commercialize compounds  -  Nov 12, 2007
FDA news (subscription),...which is in phase ii trials for fabry disease, plicera (isofagomine tartrate), which is in phase ii clinical trials for gaucher disease,

family’s unused garage converted into cool pad for teen  -  Nov 8, 2007
Knoxville News Sentinel,...who loves music and photography and plays lacrosse, has a rare genetic disorder called fabry's disease. he needs costly enzyme replacement therapy every

buyins.net: hte, nal, mpz, nbf, plx, chnr have been removed from ...  -  Nov 6, 2007
Insurance News Net (press release),...is also developing prx-102, a proprietary alpha galactosidase enzyme for the treatment of fabry disease, a genetic lysosomal storage disorder in humans,

shire human genetic therapies appoints dr. whaijen soo to manage ...  -  Nov 21, 2007
Oxfordshire Bioscience Network (press release),...usa and canada), replagal™ for the treatment of fabry disease (approved and marketed in europe) and dynepo™ for the treatment of anemia in patients with

amicus therapeutics announces third quarter 2007 financial results  -  Oct 31, 2007
Trading Markets (press release),...amigal(tm) for fabry disease and plicera(tm) for gaucher disease by yearend," stated john f. crowley, ceo of amicus therapeutics. on a reported basis,

shire goes from strength to strength with its adhd drugs  -  Nov 2, 2007
Pharma Times (subscription),...benefiting from new launches in europe. replagal (agalsidasealfa), for the treatment of fabry disease, contributed $40.7 million tothe coffers, up 26%,

shire goes from strength to strength with its adhd drugs  -  Nov 2, 2007
Pharma Times (subscription),...benefiting from new launches in europe. replagal (agalsidase alfa), for the treatment of fabry disease, contributed $40.7 million to the coffers,

further upgrade to 2007 financial guidance based on strong revenue ...  -  Nov 1, 2007
PR Newswire (press release),...with exchange rate movements against the us dollar accounting for the remaining 9% increase. human genetic therapies replagal - fabry disease sales for

Quick takes on health  -  Oct 17, 2007
Rochester Democrat and Chronicle,The session will also include a review of psychosocial services available locally to teens and children with Fabry disease and information on Fabrazyme

Daddy's Little Girl (The Update)  -  Oct 15, 2007
ProgressiveU.org,I have grown to understand the innocence in my text when exclaiming that my father, who was 47 at the time and living with Fabry's Disease, was such a

Daddy's Little Girl  -  Oct 10, 2007
ProgressiveU.org,There are a lot of symptoms of Fabry disease such as: damaging of the nerves in the body, severe depression, and/or short term memory loss, but he tries his

Diseases without clout  -  Oct 9, 2007
Checkbiotech.org (press release),Last year, the FDA approved one of Genzyme's orphan drugs: Fabrazyme, which can reverse kidney disease caused by Fabry disease, a metabolic genetic disorder

Genzyme Receives Approval to Market Elaprase(R) in Japan  -  Oct 4, 2007
CNNMoney.comThe company also markets Cerezyme(R) (imiglucerase for injection) for Type 1 Gaucher disease; Fabrazyme(R) (agalsidase beta) for Fabry disease;

My water torture hell  -  Sep 25, 2007
The Sun,But thanks to new treatment for Anderson Fabry disease – developed through trials in which George participated – he’s alive and well taking each day at a

Amicus Therapeutics announces plans to present phase 1 data for ...  -  Sep 20, 2007
Checkbiotech.org (press release),Amicus has two product candidates in Phase II clinical trials, Amigal(TM) for the treatment of Fabry disease and Plicera(TM) for the treatment of Gaucher

Genzyme expands flagship facility  -  Sep 19, 2007
In-PharmaTechnologist.com,Since then a further four products have been introduced to the facility including Fabrazyme (agalsidase beta) for Fabry disease, and Myozyme (alglucosidase

Genzyme Begins $150 Million Expansion of Boston Manufacturing Facility  -  Sep 19, 2007
Boston/SF,Three are manufactured there: Cerezyme; Fabrazyme(R) (agalsidase beta) for Fabry disease; and Myozyme(R) (alglucosidase alfa) for Pompe disease.

Shire Appoints Sylvie Gregoire President of Human Genetic ...  -  Sep 24, 2007
CNNMoney.comIn addition, REPLAGAL(TM), for the treatment of Fabry disease, which is available in Europe and other regions outside of the US, continues to perform well.

Shire deal might create 650 jobs  -  Sep 20, 2007
Boston Globe,Another of the company's drugs, Replagal, is not yet approved for use by the federal Food and Drug Administration for Fabry disease, a rare genetic disorder

Former teacher to stand trial on sex, drug charges  -  Sep 7, 2007
ChronicleHerald.ca,The Canadian Fabry Disease Initiative will evaluate the effectiveness of two forms of the only known therapy produced by different drug companies.

Policy needed  -  Sep 5, 2007
Mississauga News,Genzyme Canada makes fabrazyme for Fabry disease. I have Fabry disease, a rare genetic disorder as well as my daughter, grandson, and granddaughter.

Help for Fabry patients  -  Aug 23, 2007
SpiritIndia,...at Birmingham (UAB) have discovered the first successful treatment to stop progressive kidney function loss in patients suffering from Fabry disease.

Prescription drug cards offered  -  Sep 6, 2007
Brainerd Daily Dispatch,...immunologic disorders, Crohn's disease, Gaucher disease, pulmonary hypertension, Fabry disease, MPS 1, blood dyscrascia, growth hormone deficiency,

Combination Therapy Benefits Fabry Disease Patients  -  Aug 6, 2007
Renal and Urology News,..."This work shows for the first time that it is possible to stop progressive decline of kidney function in patients who have Fabry disease with advanced

Combination Therapy Stops Loss Of Kidney Function In Rare Genetic ...  -  Jul 27, 2007
Science Daily (press release)..."While Fabry disease is very rare, our study shows that controlling urine protein excretion will slow progression of kidney disease, even though the

Breakthrough seen for Fabry disease  -  Jul 26, 2007
United Press InternationalResearchers at the University of Alabama saw improved kidney function in 11 patients with Fabry disease, a rare genetic problem, after they were given an

Amicus Therapeutics Announces Second Quarter 2007 Financial Results  -  Aug 7, 2007
CNNMoney.com..."We expect to deliver on three significant clinical milestones by the end of 2007: Phase II data for Amigal(TM) for Fabry disease; preliminary Phase II data

Combination Therapy Halts Kidney Function Loss in Genetic Disease  -  Jul 26, 2007
MedPage Today,The treatment for Fabry's disease, an X-linked disorder caused by lysosomal-galactosidase A deficiency, carried an average yearly cost per patient of about

2006 Sales of Antibodies & Proteins  -  Aug 6, 2007
PipelineReview.com (press release),2006 sales of recombinant human enzyme products for treatment of metabolic diseases such as Gaucher’s disease, Fabry disease and Pompé disease are presented

Analysis: Street betting on Amicus' drugs  -  Jul 11, 2007
United Press InternationalHowever, the company's most advanced compound -- Amigal for Fabry disease -- is in phase 2 with data expected later this year, so it's difficult to predict

Genzyme Boosts Second-Quarter Profit, Raises Forecast (Update2)  -  Jul 25, 2007
BloombergSales of Fabrazyme, which treats Fabry disease, rose 17 percent to $104.3 million. Medicines for rare ailments account for almost half of the Genzyme's

Paladin Expands Distribution Agreement with Shire HGT to Include ...  -  Aug 8, 2007
Earthtimes.orgPaladin will be distributing ELAPRASE(TM) (idursulfase) for Hunter syndrome in addition to REPLAGAL® (agalsidase alfa) for Fabry disease.

UPDATE 2-Genzyme 2nd-qtr profit rises; raises 2007 outlook  -  Jul 25, 2007
Reuters...while sales of Fabrazyme, a treatment for Fabry disease, which causes certain fats to build up in blood vessels, rose 17 percent to $104.3 million.

Highest US Honor for Technological Innovation Awarded to Genzyme  -  Jul 26, 2007
CNW Telbec (Communiqués de presse),Fabrazyme for Fabry disease; Aldurazyme (developed with partner BioMarin Pharmaceutical) for MPS I disease; and Myozyme for Pompe disease.

Compnay receives White House honours  -  Jul 26, 2007
Mississauga News,...for Gaucher disease, fabrazyme for Fabry disease, aldurazyme (developed with BioMarin Pharmaceutical) for MPS I disease and myozyme for Pompe disease.

Genzyme Q2 Profit Rises On Strong Revenues; EPS Beats View; Lifts ...  -  Jul 25, 2007
Trading Markets,Myozyme sales, for the rare muscle disorder Pompe disease, surged to $46.7 million from $6.5 million, while Fabry disease treatment Fabrazyme sold 17% more

Zacks Broker Upgrades: Marvell, Genzyme, Praxair, Starwood Hotels  -  Jul 27, 2007
Business Wire (press release),Genzyme continues to grow its presence in the LSD franchise, based on its flagship Coenzyme product line, along with Fabrazyme (Fabry disease) and

Shire Slips To Q2 Loss On Write-off Of Assets; Non-GAAP Net Income ...  -  Jul 26, 2007
RTT News,Replagal, a long-term enzyme replacement therapy for patients with Fabry Disease, anticonvulsant drug Carbatrol and Xagrid, a treatment for a chronic

Shire plc: 2007 Guidance Upgraded as Revenue Growth Accelerates  -  Jul 26, 2007
PR Newswire (press release),REPLAGAL - Fabry disease Sales for the three months to June 30, 2007 were US$31.9 million (2006: US$28.3 million). This increase of 13% is primarily due to



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