New Data Highlighting Long-Term Efficacy and Safety Outcomes of PNH Patients Treated with Soliris® Reported at ASH Annual

  New Data Highlighting Long-Term Efficacy and Safety Outcomes of PNH Patients
  Treated with Soliris®Reported at ASH Annual Meeting

  – Large UK PNH Study Shows Significant Clinical Benefits Sustained Over 10
                                   Years –

– Two-year Data Demonstrating Long-Term Benefits of Chronic Soliris Therapy in
                     Patients with aHUS also Presented –

Business Wire

CHESHIRE, Conn. -- December 10, 2012

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced the presentation
of new data demonstrating the long-term benefits of Soliris^® (eculizumab) in
patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating,
ultra-rare and life-threatening blood disorder characterized by
complement-mediated hemolysis (destruction of red blood cells). Data including
the clinical benefits of Soliris in the treatment of patients with PNH and
atypical hemolytic uremic syndrome (aHUS) were presented at the 54th Annual
Meeting of the American Society of Hematology (ASH) in Atlanta:

  *Ten-year data from a large cohort of patients with PNH from the United
    Kingdom (UK) confirm the long-term safety and efficacy of chronic Soliris
    therapy and demonstrate the impact of Soliris on quality of life.^1
  *The long-term safety of Soliris was also demonstrated in PNH clinical
    development trials in which patients were treated continuously with
    Soliris and followed for up to 5.5 years.^2
  *Data from an international PNH registry showed that Soliris significantly
    reduced the risk of thromboembolism (TE) in patients with PNH.^3
  *Researchers from South Korea presented data confirming that hemolysis is
    an independent risk factor for TE in patients with PNH.^4
  *Researchers in Japan observed a rare genetic polymorphism in the terminal
    complement protein C5 in Japanese patients with PNH who had no or minimal
    reduction in lactate dehydrogenase (LDH) while on Soliris therapy (9 out
    of 250 treated, or 3.6%).^5
  *The ASH meeting also featured presentations of two-year data highlighting
    the long-term benefits of chronic Soliris therapy in patients with
    aHUS,^6,7 an ultra-rare, genetic disease characterized by
    complement-mediated thrombotic microangiopathy (TMA), the formation of
    blood clots in small vessels throughout the body.

Soliris, a first-in-class terminal complement inhibitor, specifically targets
uncontrolled complement activation, and is approved in the United States,
European Union, Japan and other countries as the first and only treatment for
patients with PNH. Soliris is also approved in the US and EU as the first and
only treatment for patients with aHUS.

“Extensive clinical data and real-world clinical experience spanning more than
10 years further establish the benefits that Soliris is providing to patients
with PNH,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “The
presentation of the two-year aHUS data also demonstrates the continued
benefits of long-term Soliris therapy in patients with aHUS, and underscores
the important role of hematologists in diagnosing and treating patients with
this severe and ultra-rare complement-mediated disease.”

Ten-Year Experience with Soliris in All Patients with PNH Treated in the UK

In a poster presentation today, researchers presented data from all 153
patients who participated in a nationally commissioned PNH service led by two
UK medical centers. The objective of the study was to describe the long-term
safety, efficacy, and outcomes in patients with PNH from the UK who received
Soliris treatment from May 2002 to April 2012.^1

The results demonstrated that the significant clinical benefits and long-term
safety of Soliris were sustained over 10 years of chronic treatment. The
investigators reported that long-term Soliris treatment led to an improvement
in survival when compared with historical controls, and a significant
reduction in the incidence of TE.^8,9 Researchers also reported data on the
effect of discontinuation of anti-coagulant therapy in selected PNH patients.
Transfusion independence was observed in the majority of patients, and the
number of units transfused was significantly reduced in those patients still
receiving transfusions. Results also confirmed the long-term safety and
efficacy of continuous Soliris treatment.^1

Researchers presented the following data in support of these conclusions:^1

  *Long-term Soliris therapy significantly reduced intravascular hemolysis by
    83.4%, as assessed by levels of LDH (p<0.001).
  *The researchers noted that UK patients on long-term Soliris therapy had
    improved survival compared with previously published historical
    controls.^8,9 Researchers also compared the survival of PNH patients
    treated with Soliris to a normal population of the same age and gender.
    Although the survival of PNH patients after 10 years of Soliris therapy
    was slightly inferior to this normal healthy population group, the causes
    of death in PNH patients were related to bone marrow failure and not due
    to hemolysis or TE associated with the underlying PNH.
  *In the 12 months prior to starting Soliris, 36 thrombotic episodes were
    reported in 22 patients. None of those patients had a further thrombotic
    episode while on Soliris therapy. In the most recent 12 months on therapy,
    TEs were significantly reduced with only 3 events reported in a total of 3
    patients (p<0.05).
  *Of 117 patients transfused in the 12 months before receiving Soliris, 77
    (65.8%) were transfusion-independent in the most recent 12 months on
    treatment. Among those patients still receiving transfusions (n=40), there
    was a significant 69% reduction in the number of units transfused, from a
    median of 26 units, 12 months before therapy, to 8 units in the most
    recent 12 months on therapy (P<0.05).

“More than 10 years of experience in the UK show the continuing profound
positive impact of Soliris on the lives of patients living with PNH. With
Soliris therapy, PNH-related morbidities are significantly reduced, and in
addition to this positive efficacy, there is continuing safety as well as
highly significant improvement in symptoms and quality of life,” said Anita
Hill^10, M.D. Ph.D., lead author of the poster and Consultant Haematologist at
the Leeds Teaching Hospitals, Leeds, UK.

Long-Term Safety of Sustained Soliris Treatment in Patients with PNH

In a poster presentation on December 8, researchers presented results of a
long-term safety analysis in 195 patients receiving continuous Soliris
treatment (mean duration: 30.3 months) in the Soliris PNH clinical development
trials and associated extension studies. They compared the reported incidence
of adverse events (AEs), irrespective of relation to treatment, during the
first 26 weeks of Soliris treatment with the reported incidence of AEs in the
last 26 weeks.^2

The study demonstrated the long-term safety of Soliris in PNH patients treated
for up to 5.5 years, with no evidence of cumulative toxicity. Further, the
incidence of patients reporting an AE in the last 26 weeks of treatment was
significantly reduced compared with the incidence in the first 26 weeks
(p<0.001). Additionally, the probability of a patient experiencing an AE
decreased significantly with time (p<0.001). There was also a low rate of
meningococcal infection with long-term Soliris treatment (0.422 per 100
patient-years). Two patients experienced meningococcal infections from strains
not covered by their vaccinations. Both infections were successfully treated
and both resolved without sequelae. Four patient deaths were reported over the
course of the study; none were considered by the investigators to be related
to Soliris treatment.^2

“Our data give us even greater confidence in the long-term safety of Soliris
in patients with PNH,” stated lead investigator Jeffrey Szer^11, M.D.,
Professor/Director of the Department of Clinical Haematology at Royal
Melbourne Hospital in Melbourne, Australia. “The adverse event rate among
patients on continuous Soliris therapy suggests a favorable risk-to-benefit
ratio over the long term.”

Reduced Risk of Thromboembolism (TE): An International PNH Registry Study

In a separate poster presentation today, researchers reported that Soliris is
associated with a reduced risk of TE and mortality, based on findings from
1047 patients enrolled in an international PNH registry. Over a mean follow-up
period of 22.5 months, patients receiving Soliris treatment had a cumulative
incidence of TE of 0.41% at one year and 1.35% at two years. Among patients
not taking Soliris, the corresponding one- and two-year incidences of TE were
1.70% and 2.61%, respectively. The cumulative incidence of mortality in
Soliris-treated patients was 2.31% and 4.21% at one and two years,
respectively, while in untreated patients it was 4.40% and 7.01%,
respectively. The study authors noted that results of these analyses and the
conclusions that can be drawn from them are limited due to small number of TE
and mortality outcomes.^3

“The International PNH Registry provides real-world data that are consistent
with prior research which indicated a reduced risk of thromboembolism and
mortality in patients treated with Soliris,” said Gerard Socié^12, M.D.,
Ph.D., lead author of the poster and Professor of Haematology at Hospital
Saint Louis, APHP, University Paris VII Denis Diderot, Paris, France.

Other PNH Data Presentations

The ASH meeting also featured the following data presentations in patients
with PNH:

  *In a poster presentation on December 8, researchers in South Korea
    evaluated the risk of TE in patients with PNH and elevated hemolysis (as
    identified by LDH ≥1.5 x ULN) in addition to any of the four clinical
    symptoms of abdominal pain, chest pain, dyspnea, or hemoglobinuria,
    compared with patients who had elevated LDH alone. The analyses confirm
    that elevated hemolysis was associated with a seven-fold increased risk of
    TE in patients with PNH. The risk of TE was significantly further
    increased in patients with elevated hemolysis and additional symptoms of
    abdominal pain, chest pain, dyspnea or hemoglobinuria compared with
    patients without the symptom and LDH <1.5 x ULN. These results underscore
    the importance of early therapeutic intervention and monitoring of PNH
    patients with elevated LDH.^4
  *In a separate poster presentation on December 8, data supported the need
    to test high-risk PNH patients for diagnostic markers as an aid to
    treatment selection. Researchers presented an updated analysis of 7,699
    high-risk patients whose blood cells were screened for a PNH clone using
    high-sensitivity flow cytometry. Among the 481 PNH-positive patients,
    those with large PNH clone sizes (>20%) were found to be more likely to
    have clinical symptoms, particularly those associated with hemolysis, and
    were thus deemed more likely to benefit from therapy.^13
  *A poster presentation today identified a polymorphism in the terminal
    complement protein C5 that appears to be associated with no or minimal
    reduction in LDH in nine Japanese patients with PNH who received Soliris
    therapy, although there is no evidence of the mutation outside of Japan or
    in any other Asian populations. The study authors concluded that further
    research is needed to verify that the polymorphism in the C5 gene is
    responsible for the suboptimal reduction in LDH and to determine a more
    accurate prevalence of this C5 polymorphism in Japanese populations.^5

Soliris in Patients with aHUS

During the ASH annual meeting, researchers presented two-year data that
highlight the long-term benefits of chronic Soliris therapy in patients with
aHUS. As presented last month at the annual meeting of the American Society of
Nephrology (ASN)^14, data from two pivotal phase 2 studies demonstrated that
ongoing Soliris treatment for two years was associated with sustained ongoing
inhibition of complement-mediated TMA, as indicated by a maintained increase
in platelet count, and sustained improvement in renal function and TMA
event-free status.^6,7

In a poster presented on December 8, researchers presented two-year findings
from a prospective, open-label, single-arm phase 2 trial of Soliris in adult
and adolescent patients with a long duration of aHUS and chronic kidney damage
who were undergoing prolonged plasma exchange or plasma infusion (PE/PI)
before starting treatment with Soliris. Patients had been diagnosed with aHUS
a median of 48 months prior to starting the study. Twenty patients were
enrolled in the initial study and received Soliris for 26 weeks. Nineteen of
the 20 patients continued into a long-term extension phase. Patients were
evaluated for a median duration of 114 weeks.^6

The study achieved its primary endpoint, TMA event-free status (at least 12
consecutive weeks of stable platelet count, no PE/PI, and no new dialysis), in
16 of 20 (80%) patients through 26 weeks. TMA event-free status was achieved
and maintained by 95% of patients through two years, indicating that chronic
treatment with Soliris continued to significantly inhibit complement-mediated
TMA. Patients achieved and maintained TMA event-free status regardless of the
identification of a genetic complement mutation. Importantly, no patient
required new dialysis and only one patient required any PE/PI through data
cutoff.^6

In patients treated with Soliris over two years, researchers observed
continued improvement in renal function, including a sustained improvement in
estimated glomerular filtration rate (eGFR), with a mean change from baseline
of 6.1 mL/min/1.73m^2through 26 weeks (p=0.0001) and 7.2
mL/min/1.73m^2through two years (p<0.05). Earlier intervention with Soliris
therapy was associated with greater increases in eGFR (p=0.001). Seven of 20
patients (35%) experienced chronic kidney disease (CKD) improvement of at
least one stage by 26 weeks, compared with 12 of 20 patients (60%) by two
years.^6

In a poster presented on December 9, researchers presented two-year follow-up
data from a prospective, open-label, single-arm phase 2 study in 17 adult and
adolescent patients with aHUS who had presented with progressive clinical TMA
complications despite intensive PE/PI. Patients had been diagnosed with aHUS
for a median of 10 months before the start of the study. Seventeen patients
were enrolled in the initial study and received Soliris for 26 weeks.
Seventeen patients continued into a long-term extension phase. Patients were
evaluated for a median duration of 100 weeks.^7

The study achieved its primary endpoint, as mean platelet count improved from
baseline at 26 weeks (p=0.0001). Additionally, the improved platelet count
continued over two years (p<0.001), indicating sustained inhibition of
complement-mediated TMA with ongoing eculizumab treatment. Platelet
normalization (≥150x10^9/L) was achieved in 13 of 15 patients (87%) who had
low platelets at baseline by 26 weeks and was maintained through two years for
12 of the 13 patients. TMA event-free status was also achieved rapidly and
maintained through two years, with 15 of 17 (88%) Soliris-treated patients
achieving TMA event-free status through each data cut-off point (26 weeks, one
year, and two years), and TMA event-free status was achieved regardless of the
identification of a genetic complement mutation.^7

“These longer-term data demonstrate the sustained benefits of Soliris therapy
in patients with aHUS and reveal a significant and continued improvement in
renal function over time,” commented Larry Greenbaum^15, M.D., Ph.D., Director
of Pediatric Nephrology at Emory University and Children's Healthcare of
Atlanta, who presented the aHUS poster on December 9. “Further, the data
support that earlier treatment leads to even better outcomes for patients with
aHUS.”

About PNH

PNH is an ultra-rare blood disorder in which chronic, uncontrolled activation
of complement, a component of the normal immune system, results in hemolysis
(destruction of the patient's red blood cells). PNH strikes people of all
ages, with an average age of onset in the early 30s.^16 Approximately 10% of
all patients first develop symptoms at 21 years of age or younger.^17 PNH
develops without warning and can occur in men and women of all races,
backgrounds and ages. PNH often goes unrecognized, with delays in diagnosis
ranging from one to more than 10 years.^8 In the period of time before Soliris
was available, it had been estimated that approximately one-third of patients
with PNH did not survive more than five years from the time of diagnosis.^16
PNH has been identified more commonly among patients with disorders of the
bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes
(MDS).^18-20 In patients with thrombosis of unknown origin, PNH may be an
underlying cause.^16

About aHUS

aHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic
deficiency in one or more complement regulatory genes causes chronic
uncontrolled complement activation, resulting in complement-mediated
thrombotic microangiopathy (TMA), the formation of blood clots in small blood
vessels throughout the body.^21,22 Permanent, uncontrolled complement
activation in aHUS causes a life-long risk for TMA, which leads to sudden,
catastrophic, and life-threatening damage to the kidney, brain, heart, and
other vital organs, and premature death.^21,23Sixty-five percent of all
patients with aHUS require kidney dialysis, have permanent kidney damage or
die within the first year after diagnosis despite plasma exchange or plasma
infusion (PE/PI).^24,25The majority of patients with aHUS who receive a
kidney transplant commonly experience subsequent systemic TMA, resulting in a
90% transplant failure rate in these TMA patients.^26

aHUS affects both children and adults.^27Complement-mediated TMA also causes
reduction in platelet count (thrombocytopenia) and red blood cell destruction
(hemolysis). While mutations have been identified in at least ten different
complement regulatory genes, mutations are not identified in 30-50% of
patients with a confirmed diagnosis of aHUS.^27

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from the
laboratory through regulatory approval and commercialization by Alexion.
Soliris is approved in the US, European Union, Japan and other countries as
the first and only treatment for patients with paroxysmal nocturnal
hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood
disorder, characterized by complement-mediated hemolysis (destruction of red
blood cells). Soliris is indicated to reduce hemolysis.

Soliris is also approved in the US and the European Union as the first and
only treatment for patients with atypical hemolytic uremic syndrome (aHUS), a
debilitating, ultra-rare and life-threatening genetic disorder characterized
by complement-mediated thrombotic microangiopathy, or TMA (blood clots in
small vessels). Soliris is indicated to inhibit complement-mediated TMA. The
effectiveness of Soliris in aHUS is based on the effects on TMA and renal
function. Prospective clinical trials in additional patients are ongoing to
confirm the benefit of Soliris in patients with aHUS. Soliris is not indicated
for the treatment of patients with Shiga toxinE. colirelated hemolytic
uremic syndrome (STEC-HUS).

Alexion's breakthrough approach in complement inhibition has received the
pharmaceutical industry's highest honors: the 2008 Prix Galien USA Award for
Best Biotechnology Product with broad implications for future biomedical
research, and the 2009 Prix Galien France Award in the category of Drugs for
Rare Diseases. More information, including the full prescribing information on
Soliris, is available atwww.soliris.net.

Important Safety Information

The US product label for Soliris includes a boxed warning: "Life-threatening
and fatal meningococcal infections have occurred in patients treated with
Soliris. Meningococcal infection may become rapidly life-threatening or fatal
if not recognized and treated early. Comply with the most current Advisory
Committee on Immunization Practices (ACIP) recommendations for meningococcal
vaccination in patients with complement deficiencies. Immunize patients with a
meningococcal vaccine at least 2 weeks prior to administering the first dose
of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of
developing a meningococcal infection. (See Serious Meningococcal Infections
(5.1) for additional guidance on the management of meningococcal infection.)
Monitor patients for early signs of meningococcal infections and evaluate
immediately if infection is suspected. Soliris is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy (REMS).
Under the Soliris REMS, prescribers must enroll in the program (5.2).
Enrollment in the Soliris REMS program and additional information are
available by telephone: 1-888-soliris (1-888-765-4747)."

In patients with PNH, the most frequently reported adverse events observed
with Soliris treatment in clinical studies were headache, nasopharyngitis
(runny nose), back pain and nausea. Soliris treatment of patients with PNH
should not alter anticoagulant management because the effect of withdrawal of
anticoagulant therapy during Soliris treatment has not been established. In
patients with aHUS, the most frequently reported adverse events observed with
Soliris treatment in clinical studies were hypertension, upper respiratory
tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract
infection, and leukopenia. Please see full prescribing information for
Soliris, including boxed WARNING regarding risk of serious meningococcal
infection.

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on
serving patients with severe and ultra-rare disorders through the innovation,
development and commercialization of life-transforming therapeutic products.
Alexion is the global leader in complement inhibition and has developed and
markets Soliris^®(eculizumab) as a treatment for patients with PNH and aHUS,
two debilitating, ultra-rare and life-threatening disorders caused by chronic
uncontrolled complement activation. Soliris is currently approved in more than
40 countries for the treatment of PNH, and in the United States and the
European Union for the treatment of aHUS. Alexion is evaluating other
potential indications for Soliris and is developing four other highly
innovative biotechnology product candidates, which are being investigated
across eight severe and ultra-rare disorders beyond PNH and aHUS. This press
release and further information about Alexion Pharmaceuticals, Inc. can be
found at:www.alexionpharma.com.

[ALXN-G]

Safe Harbor Statement

This news release contains forward-looking statements, including statements
related to anticipated potential health and medical benefits of Soliris®
(eculizumab) for the treatment of patients with PNH and aHUS. Forward-looking
statements are subject to factors that may cause Alexion's results and plans
to differ from those expected, including for example, decisions of regulatory
authorities regarding marketing approval or material limitations on the
marketing of Soliris for its current or potential new indications, and a
variety of other risks set forth from time to time in Alexion's filings with
the Securities and Exchange Commission, including but not limited to the risks
discussed in Alexion's Quarterly Report on Form 10-Q for the period ended
September 30, 2012, and in Alexion's other filings with the Securities and
Exchange Commission. Alexion does not intend to update any of these
forward-looking statements to reflect events or circumstances after the date
hereof, except when a duty arises under law.

    
References
      
      Hill A, Kelly R, Kulasekararaj A, et al. Eculizumab in paroxysmal nocturnal hemoglobinuria (PNH): a report of all 153 patients
1.    treated in the UK. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 10,
      2012: Abstract 3472.
      Szer J, Muus P, Roth A, et al. Long-term safety of sustained eculizumab treatment in patients with paroxysmal nocturnal
2.    hemoglobinuria. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 8, 2012:
      Abstract 1260.
      Socié G, Schrezenmeier H, Muus P, et al. Eculizumab protects against TE and prolongs survival in patients with paroxysmal nocturnal
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      (ASH), Atlanta, GA, December 10, 2012: Abstract 3480.
      Lee JW, Jang JH, Kim JS, et al. Risk of thromboembolism in patients with paroxysmal nocturnal hemoglobinuria presenting with both
4.    clinical symptoms and elevated hemolysis. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH),
      Atlanta, GA, December 8, 2012: Abstract 1273.
      Nishimura J, Yamamoto M, Hayashi S, et al. A rare genetic polymorphism in C5 confers poor response to the anti-C5 monoclonal
5.    antibody eculizumab by nine Japanese patients with PNH. Presented at the 54th Annual Meeting of the American Society of Hematology
      (ASH), Atlanta, GA, December 10, 2012: Abstract 3197.
      Licht C, Muus P, Legendre C, et al. Eculizumab (ECU) safety and efficacy in atypical hemolytic uremic syndrome (aHUS) patients with
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      Greenbaum L, Legendre C, Babu S, et al. Eculizumab (ECU) in atypical hemolytic uremic syndrome (aHUS) patients with progressing
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10.   Dr. Anita Hill receives research support from Alexion Pharmaceuticals, Inc. and has served on advisory boards for the company.
11.   Dr. Jeffrey Szer has no relevant conflicts of interest to disclose.
12.   Dr. Gerard Socié has no relevant conflicts of interest to disclose.
      Movalia MK, Illingworth A. Distribution of PNH clone sizes within high risk diagnostic categories among 481 PNH positive patients
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      http://news.alexionpharma.com/press-release/product-news/two-year-data-show-long-term-benefits-chronic-soliris-therapy-patients-ah.
15.   Dr. Larry Greenbaum receives research support from Alexion Pharmaceuticals, Inc. and is a consultant to the company.
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      cells in bone marrow failure syndrome. Blood. 2002;100 (12):3897-3902.
19.   Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal haemoglobinuria clones in patients with myelodysplastic syndromes.
      Br J Haematol. 1998;102(2):465-474.
20.   Maciejewski JP, Rivera C, Kook H, Dunn D, Young NS. Relationship between bone marrow failure syndromes and the presence of
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21.   Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin Nephrol Hypertens. 2010;19(3):242-7.
22.   Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic
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23.   Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int. 2006;70(1):16-23.
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      and outcome. Blood. 2006;108:1267-1269.
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27.   Noris M, Caprioli J, Bresin E, et al. Relative role of genetic complement abnormalities in sporadic and familial aHUS and their
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Contact:

Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Executive Director, Corporate Communications
or
Media:
Alexion Pharmaceuticals, Inc.
Kim Diamond, 203-439-9600
Director, Corporate Communications
or
Investors:
Rx Communications
Rhonda Chiger, 917-322-2569
 
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