The U.S. Food and Drug Administration today approved Elelyso (taliglucerase alfa) for long-term enzyme replacement therapy to treat a form of Gaucher disease, a rare genetic disorder.

Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials (lipids) to collect in the spleen, liver, kidneys, and other organs. The major signs of Gaucher disease include liver or spleen damage, low red blood cell counts (anemia), low blood platelet counts, and bone problems.

Elelyso is an injection that replaces the missing enzyme in patients with a confirmed diagnosis of Type 1 (non-neuropathic) Gaucher disease and should be administered by a health care professional every other week. Type 1 Gaucher disease is estimated to affect about 6,000 people in the United States.

“Today’s approval provides for a new enzyme replacement therapy for the select number of patients with Type 1 Gaucher disease,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research. “It also demonstrates FDA’s commitment to developing treatments for rare diseases.”

Due to the small number of affected patients, the efficacy of Elelyso was evaluated in a total of 56 patients with Type 1 Gaucher disease enrolled in two clinical trials. Many of these patients continued treatment on a longer-term extension study.

In one multi-center, double-blind, parallel-dose trial, the efficacy of Elelyso for use as an initial therapy was evaluated in 31 adult patients who had not previously received enzyme replacement therapy. Patients were randomly selected to receive Elelyso at a dose of either 30 units per kilogram or 60 units/kg.

At both doses, Elelyso was effective in reducing spleen volume, the study’s primary endpoint, from baseline by an average of 29 percent in patients receiving the 30 units/kg dose and by an average of 40 percent in patients receiving the 60 units/kg dose after nine months of treatment. Improvements in liver volume, blood platelet counts, and red blood cell (hemoglobin) levels also were observed.

In the other study, the efficacy of Elelyso was assessed in 25 patients with Type 1 Gaucher disease who were switched from imiglucerase, another enzyme replacement therapy product. In this multi-center, open-label, single-arm trial, patients who had been receiving treatment with imiglucerase for at least two years were switched to Elelyso infusions every other week at the same dose of imiglucerase. Results showed Elelyso was effective in maintaining spleen and liver volumes, blood platelet counts, and hemoglobin levels over a nine month evaluation period.

The most common side effects reported during clinical studies were infusion reactions and allergic reactions. Symptoms of infusion reactions include headache, chest pain or discomfort, weakness, fatigue, hives, skin redness, increased blood pressure, back pain, joint pain, and flushing. As with other intravenous protein products, anaphylaxis has been observed in some patients during Elelyso infusions.

Other commonly observed side effects observed in greater than 10 percent of patients treated with Elelyso included upper respiratory tract infection, common cold-like symptoms (nasopharyngitis), joint pain (arthralgia), influenza, headache, extremity pain, back pain, and urinary tract infections.

Elelyso is manufactured and distributed by New York City-based Pfizer Inc., under license from Protalix BioTherapeutics Inc.

...and will continue to be a priority in the future, we have pledged to help any Gaucher patient who ask us for help where ever they live. However the “Gaucher-world” has changed over the last couple of years with the introduction of new therapies and new companies coming into the market and it is necessary to look at how best to approach the provision of humanitarian aid to ensure sustainability and security for patients and their families.

For this reason a very important meeting took place in Amsterdam at the Amsterdam Medical Centre on the 5th of October 2011, convened by the EWGGD and EGA. Read more...

European Medicines Agency Approval Adds Significant Capacity for the Manufacture of Shire’s Enzyme Replacement Therapies

Lexington, MA, US – February 22, 2012 – Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use has approved the production of VPRIV® (velaglucerase alfa) in its new state-of-the-art manufacturing facility at 400 Shire Way in Lexington, MA. The European Commission’s decision is expected imminently.

“We welcome the news that Shire's new manufacturing facility in Lexington has received EMA approval for the production of VPRIV,” said Tanya Collin-Histed of the European Gaucher Alliance.  “This provides patients with greater comfort over the maintenance of supply of enzyme therapies for the treatment of Gaucher disease.” 

Shire now has two EMA approved facilities – Alewife, in Cambridge, MA, as well as the new Lexington facility – in which to manufacture VPRIV drug substance. This additional capacity will allow Shire to significantly increase global supply of VPRIV and provides additional manufacturing flexibility. The EMA approval is also a critical first step in releasing further capacity for the manufacturing of REPLAGAL® (agalsidase alfa) at Shire’s Alewife facility. The new facility increases bioreactor capacity from 1000 to 8000L, and is the first commercially licensed facility in the world to utilize single-use bioreactor and disposable technology throughout cell culture processing to reduce manufacturing risk.

“I am delighted to announce the EMA approval of our facility. Shire has invested strategically in new manufacturing facilities and state-of-the-art technology because we recognize the critical importance of ensuring the continuity of treatment for patients with rare and life-threatening diseases," said Bill Ciambrone, Senior Vice President, Technical Operations, Shire HGT. “The EMA approval of VPRIV in this manufacturing plant, only three years after breaking ground, is a testament to the hard work and dedication of Shire employees, and represents crucial additional capacity for manufacturing our enzyme replacement therapies for Gaucher and Fabry patients.”

About The 400 Shire Way Manufacturing Facility
Shire has invested over $200M in manufacturing infrastructure and technology to ensure that we maintain a reliable and consistent drug supply. In keeping with Shire’s corporate sustainability commitments, this new manufacturing plant has met the requirements for Leadership in Energy and Environmental Design (LEED) Certification and will receive formal recognition from the United States Green Building Council this quarter. In addition to increasing capacity and reducing manufacturing risk, utilization of single-use technology requires approximately 80% less water and 50% less energy than a conventional manufacturing plant.

About VPRIV (velaglucerase alfa)
VPRIV is made using Shire’s gene activation technology, in a human cell line. The enzyme produced has the exact human amino acid sequence as that found in the naturally occurring human enzyme.

VPRIV is used for the long-term treatment of patients with type 1 Gaucher disease.

VPRIV is approved in 38 countries globally, including the US, the European Union, and Israel and is for patients who are treatment-naïve as well as patients who have previously been treated with imiglucerase.

About REPLAGAL (agalsidase alfa)
REPLAGAL is a human form of enzyme alpha-galactosidase A (α-Gal A) manufactured in a human cell line by gene activation. 2011 marked the 15th year of clinical experience with REPLAGAL, which is now approved in 46 countries worldwide. REPLAGAL is not currently approved for commercial sale in the U.S.
REPLAGAL is the only human-cell-line-derived form of enzyme replacement therapy that is indicated for the long-term treatment of patients with a confirmed diagnosis of Fabry disease (α-Gal A deficiency).

VPRIV Important Safety Information
The most serious adverse reactions seen with VPRIV were hypersensitivity reactions. Infusion-related reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of infusion-related reactions were: headache, dizziness, low or high blood pressure, nausea, tiredness and weakness, and fever.  Generally the infusion-related reactions were mild and, in treatment-naïve patients, onset occurred mostly during the first 6 months of treatment and tended to occur less frequently with time.  
All adult side effects of VPRIV are considered relevant to children (ages 4 to 17 years). Side effects more commonly seen in children compared with adult patients included: upper respiratory tract infection, rash, aPTT prolonged, and fever. The safety of VPRIV has not been established in patients younger than 4 years of age.
VPRIV is not available in all countries and prescribing information may differ between countries.  Please consult your local prescribing information. Full prescribing information for VPRIV in the U.K. can be found at www.VPRIV.co.uk  

REPLAGAL Important Safety Information
The most serious adverse reactions seen with REPLAGAL were hypersensitivity reactions. Infusion-related reactions were the most commonly observed adverse reactions in patients treated with REPLAGAL in clinical studies. Most side effects are mild to moderate and include headache, tingling, numbness, tremors, fatigue, change in temperature sensation, increased blood pressure, upset stomach, diarrhea, coughing, sore throat, difficulty sleeping, change in the taste of food, change in smell, difficulty speaking, acne, dry skin and eye problems. About 1 out of 10 patients may have a reaction during or shortly after infusion of REPLAGAL. These effects include chills and facial flushing (warmth and redness).

REPLAGAL is not available in all countries and prescribing information may differ between countries.  Please consult your local prescribing information.

For further information please contact:

Investor Relations Eric Rojas (erojas@shire.com)
Sarah Elton Farr (seltonfarr@shire.com)
+1 781 482 0999
+44 1256 894 157

Media 
Jessica Mann (jmann@shire.com)

+44 1256 894 280
 Jessica Cotrone (jcotrone@shire.com)
+1 781 482 9538

Notes to editors

SHIRE PLC

Shire’s strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI) diseases; regenerative medicine as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire’s in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.

For further information on Shire including the SmPC, please visit the Company’s website: www.shire.com.

"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995

Statements included herein that are not historical facts are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent uncertainty of research, development, approval, reimbursement, manufacturing and commercialization of Shire’s Specialty Pharmaceuticals, Human Genetic Therapies and Regenerative Medicine products, as well as the ability to secure new products for commercialization and/or development; government regulation of Shire’s products; Shire’s ability to manufacture its products in sufficient quantities to meet demand; the impact of competitive therapies on Shire’s products; Shire’s ability to register, maintain and enforce patents and other intellectual property rights relating to its products; Shire’s ability to obtain and maintain government and other third-party reimbursement for its products; and other risks and uncertainties detailed from time to time in Shire’s filings with the Securities and Exchange Commission

Genzyme, a Sanofi company, announced today four-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate. Sustained or further improvements were observed across all endpoints, including markers of bone disease, at the four-year timepoint. The results will be presented for the first time this week at the Lysosomal Disease Network WORLD Symposium in San Diego, Calif.

Eliglustat tartrate, a capsule taken orally, is being developed to provide a convenient treatment alternative for adult patients with Gaucher disease type 1, and to offer a broader range of treatment options for patients and physicians to achieve individual therapeutic goals. Genzyme’s Cerezyme®
(imiglucerase for injection), the standard of care for patients with Gaucher disease type 1, is administered through intravenous infusions.

Genzyme previously reported that the 52-week phase 2 trial of eliglustat tartrate had met its primary composite endpoint: a clinically meaningful response in at least two of three endpoints (improvements in spleen size, hemoglobin and platelet levels) in individual patients. Patients have continued to receive eliglustat tartrate in the extension portion of the study for over four years. The data from patients on eliglustat after four years indicate continued or stabilized improvements across all endpoints:
 Spleen and liver volumes decreased from baseline by a mean of 63 percent and 28 percent respectively.
 Hemoglobin and platelet levels had increased from baseline by a mean of 2.3 g/dL and 95 percent respectively.
 All patients (100 percent) had met at least three of the four hematologic and visceral therapeutic goals established for enzyme replacement therapy
 These data also indicate continued improvement in bone mineral density by DXA, with a mean Tscore increase of 0.8 from baseline in the lumbar spine.

“Eliglustat tartrate represents a new hope for Gaucher disease type 1 patients,” said Manisha Balwani, MD, MS, Department of Genetics and Genomic Sciences, Mount Sinai School of Medicine. “The option of an oral therapy offers convenience, expanded access to treatment and, most important, an improvement in the quality of life for patients. Eliglustat marks a potential paradigm shift in Gaucher treatment.”

“The four-year data from our phase 2 eliglustat study show very encouraging results, particularly the continued improvements observed in markers of bone disease," said Genzyme’s Head of Rare Diseases, Rogerio Vivaldi, M.D. “The efficacy of eliglustat tartrate, combined with its safety profile, demonstrate its potential to transform the treatment experience for the Gaucher community. We look forward to continuing our momentum in the phase 3 program.”

In the phase 2 study, the most common adverse events reported in greater than two patients through four years of treatment included viral infections (six patients), urinary tract and upper respiratory tract infections (four patients each), and nasopharyngitis, sinusitis, arthralgia, pain in extremity, headache, 2/3 increased blood pressure, abnormal nerve conduction study, abdominal pain, and diarrhea (three patients each). Ten drug-related adverse events, including one serious event, were reported in eight patients. All related events were mild in severity.

Genzyme has also fully enrolled all three phase 3 trials for the oral therapy. Combined, these trials represent the largest clinical program ever focused on Gaucher disease, with participating sites in over 30 countries. In total, more than 350 patients are enrolled in the phase 3 studies.

The first phase 3 trial, ENCORE, is a randomized, open-label study for adult patients with Gaucher disease type 1, designed to compare eliglustat tartrate to Cerezyme. Adult patients who previously received enzyme replacement therapy for at least three years and have reached their therapeutic goals are enrolled in this trial.

The second trial, ENGAGE, is a randomized, double-blind, placebo-controlled study for patients with Gaucher disease type 1 who were untreated or had not been on treatment for at least nine months prior to study entry.

Data from these pivotal registration studies are expected in the first half of 2013. A third trial, known as EDGE, compares once-daily dosing of eliglustat tartrate with twice-daily dosing.

Protalix BioTherapeutics, Inc. announced today that new clinical data on taliglucerase alfa will be presented at the 8th Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2012 being held February 8-10 in San Diego, California. Taliglucerase alfa is the Company's proprietary plant cell expressed recombinant form of human Glucocerebrosidase (GCD), which is being developed for the treatment of Gaucher disease.

Professor Ari Zimran, M.D., Director of the Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel, is presenting long-term safety and efficacy data from the Company's double-blind, follow-on extension study of taliglucerase alfa for the treatment of Gaucher disease in naive patients. Eligible patients who completed nine months of treatment in the Company's pivotal phase III clinical trial were offered the opportunity to participate in the extension study and continue to receive taliglucerase alfa at the same dose they received in the phase III clinical trial for an additional 15 months in a blinded manner. Accordingly, the extension trial included two treatment groups; one treated with a 60 U/kg dose and the other with a 30 U/kg dose. The major endpoints of the study were spleen volume, liver volume, hemoglobin concentration, platelet count, and chitotriosidase activity. Twenty-six patients were enrolled in the extension trial which was performed in centers throughout Europe, Israel, North America, South America and South Africa.

Patients treated with taliglucerase alfa in the extension trial continued to demonstrate a statistically significant reduction in mean spleen volume after 24 months, compared with baseline, in both treatment groups. Both dosage groups demonstrated statistically significant mean reductions in spleen volume; reductions of 54.0% (p<0.0001) in the 60 U/kg group and of 41.0% (p<0.0001) in the 30 U/kg group. Statistically significant improvements were also observed in all secondary end points. Both groups demonstrated statistically significant mean reductions in liver volume; reductions of 17.5% (p<0.0003) in the 60 U/kg group and of 20.6% (p<0.0001) in the 30 U/kg group. Patients with Hepatomegaly demonstrated a reduction in liver volume of 25.2% (p<0.0001).
Statistically significant mean increases in hemoglobin concentration were also demonstrated by both groups; mean increases from baseline (from 11.6 g/DL to 13.9 g/DL (p<0.0020)) (25.8%) in the 60 U/kg group and mean increases from baseline (from 12.4 g/DL to 13.6 g/DL (p<0.0314)) (13.2%) in the 30 U/kg group. Anemic patients demonstrated a mean increase from baseline in hemoglobin concentration (from 9.5 g/DL to 12.9 g/DL (p<0.0013)) (40.8%).

The data also demonstrated statistically significant mean increases in platelet count for both groups; increases from 69,043 to 141,071 (p<0.0016) in the 60 U/kg group and from 64,900 to 93,333 (p<0.0105) in the 30 U/kg group.

Lastly, statistically significant mean reductions in chitotriosidase activity were demonstrated by both groups; a reduction of 76% (p<0.0001)) in the 60 U/kg group and of 61% (p<0.0001) in the 30 U/kg group.

"Pivotal and follow-up clinical studies of taliglucerase alfa to date demonstrate that taliglucerase alfa may be an effective treatment for Gaucher disease," said Dr. Ari Zimran. "The results of this 24 month extension trial suggest that taliglucerase alfa has the potential to become a treatment alternative for Gaucher disease patients should it be approved."

The safety analysis presented for both treatment groups demonstrates that taliglucerase alfa was well tolerated, and no drug related serious adverse events were reported. Two patients who participated in the extension trial developed neutralizing IgG antibodies that were determined to be positive in an in vitro assay, and were determined to be negative in a cell-based assay. In addition, one patient in the 60 U/kg dose group experienced a hypersensitivity reaction during month 10 of treatment. Treatment of this patient has been continued with premedication for an additional 32 months without any treatment related adverse event reported.

Gregory Pastores, M.D., Professor of Neurology and Pediatrics and Director of the Neurogenetics Laboratory at the New York University School of Medicine, is presenting at the WORLD 2012 Symposium the full results of all adult patients that participated in the Company's multi-center, open-label, nine month switchover trial of taliglucerase alfa for the treatment of Gaucher disease. In the switchover trial, patients with stable disease were switched from treatment via intravenous infusions of imiglucerase (Cerezyme (R)) to intravenous infusions of taliglucerase alfa every two weeks at an equivalent dose to the patient's previous imiglucerase dose. Twenty-six adult patients were enrolled in the switchover trial which was performed in centers throughout Europe, Israel, North America and Australia.

The results of the switchover trial demonstrate that over a nine-month treatment period of the study, patients remained stable with regard to the efficacy endpoints--spleen volume, liver volume, platelet count and hemoglobin concentration--after switching to taliglucerase alfa from imiglucerase. The safety analysis presented for the switchover trial demonstrates that taliglucerase alfa was well tolerated, and no drug related serious adverse events were reported. One patient developed neutralizing IgG antibodies that were determined to be positive in an in vitro assay, and were determined to be negative in a cell-based assay. Another patient experienced a hypersensitivity reaction, which was treated in a physician's office and resolved. The patient declined to continue infusions with premedication.

"The current findings are in accord with the interim observations made last year, which revealed maintenance of clinical benefit in patients switched from imiglucerase to taliglucerase across all doses. Additionally, all drug-related adverse effects were mild or moderate and transient in nature," said Dr. Gregory Pastores.

Laura van Dussen, M.D., of the Academic Medical Center, University of Amsterdam, is presenting long term bone marrow responses, as measured by Quantitative Chemical Shift Imaging (QCSI) MRI, following treatment of Gaucher disease patients with taliglucerase alfa. Eight patients from the Company's pivotal and extension trial had their fat fraction evaluated by QCSI. At 24 months, seven of the eight patients demonstrated significant improvement in fat fraction from baseline. One patient remained unchanged. In addition, four patients whose score for bone was determined to be "at risk" at baseline (fat fraction <0.23) were no longer classified as "at risk" according to the protocol after 24 months of treatment with taliglucerase alfa.

Marketing applications for taliglucerase alfa have been filed in the United States, Europe, Israel, Brazil and Australia. The U.S. Food and Drug Administration Prescription Drug User Fee Act (PDUFA) target action date for taliglucerase alfa is May 1, 2012.

I would like to thank you all who attended the first regional meeting of Gaucher patients, on 5th November 2011, in Čatež, Slovenia. I would also like to thank the colleagues from Genzyme for logistical and financial support.

More than 50 people from six countries (Slovenia, Croatia, Bosnia & Herzegovina, Macedonia, Bulgaria and Italy) attended the meeting:

- 10 Gaucher patients from Slovenia, i.e. more than 50 % of all Slovenian Gaucher patients,

- Ms Vlasta Zmazek, who as a chairman of Croatian Rare Disease Association represented Croatian Gaucher patients,

- 2 Gaucher patients from Bosnia and Herzegovina,
- 1 Gaucher patient from Macedonia,
- Ms Radoslava Tomova, board member of European Gaucher Alliance (EGA), Gaucher patient and a chairman of Bulgarian Gaucher Association, 
- Mr Borislav Đurić, a chairman of  Rare Disease Association of Bosnia and Herzegovina,
- 5 physicians who treat Gaucher patients,
- 3 nurses who, among other duties, teach Gaucher patients how to perform home therapy,

- family members and friends of Gaucher patients,
- 6 Genzyme representatives,
- 2 journalists.

This was the first regional meeting of Gaucher patients in this part of Europe and for some also the first opportunity to meet other Gaucher patients. It is of high value that we exchanged our experiences, met each other and learned a lot. I would like to thank Dr Samo Zver, Dr Polona Novak, Dr Lidija Kitanovski, Dr Majda Benedik Dolničar, Dr Bruno Bembi, Dr Toni Valković and Ms Branka Založnik for their excellent presentations on different aspects of Gaucher disease and its treatment. My thanks go also to Ms Radoslava Tomova and Ms Vlasta Zmazek for the presentations of organisations that represent the voice of Gaucher patients. Thanks too go to Dr Vukašin Andrić for the presentation of Genzyme company, as well as of Cerezyme production process. I am glad that many participants were asking questions and took an active part in discussion.

All who filled in the questionnaire have found the meeting interesting and would like to attend such a meeting also in the future. I am grateful for all your suggestions and responses written in the questionnaire leaflets.  Please feel free to send your further suggestions regarding future meetings or other activities of our Association to my e-mail or to info@gaucher-drustvo.si. This is also the address for all your inquiries regarding Gaucher disease. Additionally, I would like to invite you to visit the new European Gaucher Alliance (EGA) website: www.eurogaucher.org.

Irena Žnidar

Spoštovani,

Hvala vsem, ki ste se udeležili prvega regijskega srečanja gaucherjevih bolnikov, ki je potekalo 5. novembra 2011 v Čatežu. Hvala kolegom iz Genzyma za pomoč pri organizaciji in za financiranje srečanja.

Srečanja se je udeležilo več kot 50 ljudi iz šestih držav (Slovenija, Hrvaška, BiH, Makedonija, Bolgarija, Italija):
- 10 gaucherjevih bolnikov iz Slovenije, tj. več kot polovica vseh slovenskih gaucherjevih bolnikov,
- gospa Vlasta Zmazek, ki je kot predsednica Hrvaškega združenja za redke bolezni predstavljala gaucherjeve bolnike iz Hrvaške,
- 2 gaucherjevi bolnici iz Bosne in Hercegovine,
- 1 gaucherjeva bolnica iz Makedonije,
- gospa Radoslava Tomova, članica odbora Evropske zveze Gaucher (EGA), gaucherjeva bolnica in predsednica bolgarskega društva gaucherjevih bolnikov, 
- gospod Borislav Đurić, predsednik Združenja za redke bolezni BiH,
- 5 zdravnikov, ki zdravijo gaucherjeve bolnike,
- 3 medicinske sestre, ki med drugim usposabljajo gaucherjeve bolnike za infundiranje zdravila na domu,
- družinski člani in prijatelji gaucherjevih bolnikov,
- 6 predstavnikov Genzyma,
- 2 novinarki.

To je bilo prvo tovrstno srečanje v tem delu Evrope in za nekatere celo prva priložnost spoznati druge gaucherjeve bolnike. Dragoceno je, da smo si lahko izmenjali izkušnje, se spoznali med seboj ter se marsikaj novega naučili. Zahvaljujem se dr. Samu Zveru, dr. Poloni Novak, dr. Lidiji Kitanovski, dr. Majdi Benedik Dolničar, dr. Brunu Bembiju, dr. Toniju Valkoviću in s. Branki Založnik za izvrstne prezentacije različnih vidikov gaucherjeve bolezni in njene obravnave. Hvala gospema Radoslavi Tomovi in Vlasti Zmazek za predstavitev organizacij, kamor smo vključeni gaucherjevi bolniki. Hvala dr. Vukašinu Andriću za predstavitev podjetja Gezyme in predstavitev poteka proizvodnje zdravila cerezyme. Vesela sem, da je veliko prisotnih sodelovalo s svojimi vprašanji in diskusijo.

Vsem prisotnim, ki ste izpolnili vprašalnik, je bilo srečanje všeč in si še želite podobnih srečanj. Zahvaljujem se za vaše predloge in odzive, ki ste jih zapisali na vprašalnik. Vaše predloge in ideje glede prihodnjih srečanj, kot tudi glede drugih aktivnosti našega društva, lahko kadarkoli pošljete na moj naslov ali na info@gaucher-drustvo.si. To je tudi naslov za vse, kar vas zanima v zvezi z gaucherjevo boleznijo. Vabljeni tudi k ogledu nove spletne strani Evropske zveze Gaucher (EGA): www.eurogaucher.org.

V upanju da se kmalu ponovno vidimo, vas vse lepo pozdravljam,

Irena Žnidar

This launch takes place after several months of hard work by EGA Directors Tanya Collin-Histed and Pascal Niemeyer who have worked closely with Score Communications based in London, UK.
Special thanks to Teresa, from the Spanish Gaucher Association, as well as to Fern, from the Italian Gaucher Association, for the translation into their languages.

We hope you will like the new website and  would welcome your feedback by sending an email to:

info@eurogaucher.org 

Please do not hesitate to send us any suggestions for improvement or details about missing information.

CETP will be reinstated now due to prolonged shortage of Cerezyme supplies and based on a recommendation of the European Medicine Agency (EMA).

Recommendations by EWGGD for monitoring during imiglucerase reduction or cessation of enzyme replacement therapy and during the recovery period:

Application process:

CETP board members:

Program coordinator and assistant:

CETP coordinating centre contact details: