Keep yourself updated with HAEi news and events

HAEi News Archive

25Mar, 2017

Japan Approves Berinert for Short-Term Prophylaxis

As of 24 March 2017, Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved an extended use of Berinert [...]

23Mar, 2017

Pivotal Phase III Data for CSL Behring’s Subcutaneous C1-Esterase Inhibitor

New England Journal of Medicine (NEJM) has published results from the COMPACT study, a pivotal Phase III study evaluating the [...]

22Mar, 2017

Cooperation and networking among health care professionals worldwide

From 7 to 9 March 2017, CSL Behring in Bern, Switzerland hosted the First Intercontinental Immunoglobulin and Hereditary Angioedema Academy. [...]

16Mar, 2017

Cinryze first HAE treatment approved for routine prevention in paediatrics

The European Commission has approved a label extension granting three new indications for Cinryze (C1 inhibitor [human]) from Shire plc, [...]

10Mar, 2017

Financial report 2016 from Pharming Group

Pharming Group N.V. has presented its (unaudited) financial report for the full year ended 31 December 2016. Sijmen de Vries, [...]

10Mar, 2017

KalVista appoints Senior Vice President of Medical

KalVista Pharmaceuticals, Inc. has appointed Andreas Maetzel, M.D., M.Sc., Ph.D, to the role of Senior Vice President, Medical. “Andreas Maetzel [...]

8Mar, 2017

Attune Pharmaceuticals announces pre-clinical data

Attune Pharmaceuticals, a biotechnology company focused on the discovery and development of novel oral small molecule therapeutics for the treatment of [...]

28Feb, 2017

Positive interim results from APeX-1 trial

BioCryst Pharmaceuticals, Inc. has announced results from an interim analysis of its Phase 2 APeX-1 trial in HAE. APeX-1 is a dose [...]

23Feb, 2017

Phase 1b results for investigational treatment published

The New England Journal of Medicine has just published the results from the Phase 1b study of lanadelumab (SHP643; formerly DX-2930) [...]

17Jan, 2017

European Commission amends Marketing Authorisation for Ruconest

Following the positive opinion of the Committee for Medicinal Products for Human Use (CHMP), the European Commission has adopted the [...]

24Dec, 2016

Global Perspectives – the HAEi magazine #1/2016 is out now

We are excited to inform you that the first edition of "Global Perspectives" - the HAEi magazine - is out now. Please download [...]

23Nov, 2016

KalVista Closes Merger with Carbylan

KalVista Pharmaceuticals, Inc. has announced the closing of the previously announced merger with Carbylan Therapeutics, Inc. As a result of the [...]

17Nov, 2016

A Randomized Trial of Subcutaneous C1-INH for HAE Attack Prevention

Current options for prophylaxis of acute HAE attacks are very limited. The international, prospective, double-blind, crossover, phase III COMPACT trial [...]

15Nov, 2016

Results of phase II study for prophylaxis of HAE attacks

The results of a “Randomized, Double-Blind, Placebo-Controlled Trial of Recombinant Human C1 Inhibitor for Prophylaxis of Hereditary Angioedema Attacks” has been [...]

15Nov, 2016

Pharming announces progress on financing

Pharming Group N.V. has made significant progress with financing towards completion of the transaction with subsidiaries of Valeant Pharmaceuticals International, [...]

11Nov, 2016

CHMP adopts positive opinion to include self-administration for Ruconest

The Committee for Medicinal Products for Human Use (CHMP), an advisory committee of the European Medicine Agency (EMA), has adopted [...]

28Oct, 2016

Financial report from Pharming

Pharming Group N.V. presents its financial report for the nine months ended 30 September 2016. From CEO Sijmen de Vries' comments: [...]

12Sep, 2016

HAEi Newsletter #4/2016 out now

We are excited to inform you that the fourth edition of the 2016 newsletter is out now. Again, the newsletter offers our [...]

31Aug, 2016

FDA Accepts Application for Subcutaneous Prophylactic Therapy

The U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for CSL Behring's low-volume [...]

11Aug, 2016

KalVista Initiates Phase 1 Clinical Trial for HAE Treatment

KalVista Pharmaceuticals announces the dosing of the first subject in a first-in-human clinical trial to evaluate the safety, pharmacokinetics and [...]

11Aug, 2016

BioCryst Initiates APeX-1 Clinical Trial of BCX7353 for HAE

BioCryst Pharmaceuticals, Inc. has dosed the first subject in the APeX-1 clinical trial of BCX7353 for the oral treatment of [...]

9Aug, 2016

Pharming acquire all North American commercialization rights to Ruconest

Pharming Group N.V. has entered into a definitive agreement to acquire all North American commercialization rights to its own product [...]

5Aug, 2016

BioCryst continues to make progress

Announcing the BioCryst Pharmaceuticals financial results for the second quarter of 2016 Jon P. Stonehouse, President & CEO, said: "We continue to [...]

19Jul, 2016

U.S. FDA Approves Berinert as the First and Only Pediatric Treatment

The U.S. Food and Drug Administration (FDA) has approved the use of Berinert [C1 Esterase Inhibitor (Human)], the CSL Behring therapy for treating [...]

18Jul, 2016

Pharming Announces Positive Results from Trial with Ruconest

Pharming Group N.V. has announced positive results from a Phase 2 clinical study of Ruconest (recombinant C1 esterase inhibitor, 50 IU/kg) [...]

15Jul, 2016

Pharming amends Ruconest Distribution Agreement with Sobi

Pharming Group NV and Swedish Orphan Biovitrum AB (Sobi) has signed an amendment of their Ruconest distribution agreement from 2009. In addition [...]

22Jun, 2016

HAEi Newsletter #3/2016 out now

We are excited to inform you that the third edition of the 2016 newsletter is out now. Again, the newsletter offers our [...]

16Jun, 2016

Carbylan and KalVista Enter into Share Purchase Agreement

Carbylan Therapeutics, Inc. and KalVista Pharmaceuticals Ltd. have entered into a definitive share purchase agreement pursuant to which the shareholders [...]

2Jun, 2016

12,000,000+ steps for HAE

Before hae day :-) 2016 quite a number of people told HAEi that they would really have liked to take [...]

26May, 2016

80 people walked the Camino for HAE

From 15 to 17 May 2016 almost 80 people – the majority of them suffering from HAE – walked part [...]

19May, 2016

KalVista to Support Global HAE Conference

KalVista Pharmaceuticals supports the third HAE Global Conference in Madrid, Spain, from 19-22 May 2016. Andrew Crockett, KalVista’s CEO, said: [...]

19May, 2016

Change to Pharming strategy now bearing fruit

From the Pharming Group N.V. financial report for the first quarter ended 31 March 2016: During the quarter we initiated [...]

17May, 2016

CSL Behring Commemorates 2016 hae day :-)

CSL Behring joins the global HAE community today in recognition of the fifth annual hae day :-), an initiative led [...]

17May, 2016

Pharming Group Supports HAE DAY :-) 2016

Pharming Group N.V. supports the Fifth Annual International Hereditary Angioedema Day, hae day :-) taking place on 16 May 2016 and aiming [...]

6May, 2016

Working through the start-up activities

  "We are currently working through the start-up activities for the APeX-1 trial of BCX7353 for prevention of angioedema attacks [...]

1May, 2016

HAEi Newsletter #2/2016 out now

We are excited to inform you that the second edition of the 2016 newsletter is out now. Again, the newsletter offers our [...]

27Apr, 2016

Join the global HAE Walk

  Quite a number of people have told HAEi that they would really have liked to be with us in [...]

22Apr, 2016

Raising awareness step by step on the Camino

80 people – most of them suffering from a rare and potentially fatal disease – will meet mid-May in northern [...]

8Apr, 2016

Change in Marketing Authorization Terms For Ruconest

The European Commission has adopted the CHMP recommendation to include the treatment of HAE attacks in adolescents with HAE and [...]

30Mar, 2016

Shire 2015 Annual Report

HAE related information from the Shire plc 2015 Annual Report: The $6 billion acquisition of Dyax expands and extends our [...]

30Mar, 2016

Global Blood Therapeutics Reports Recent Business Progress

Global Blood Therapeutics, Inc., a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet [...]

24Mar, 2016

Cinryze now available in Canada

Cinryze is now available in Canada to patients with HAE who may benefit from routine prevention. It is the first and only treatment [...]

12Mar, 2016

80 people to walk the Camino for HAE

For the 2016 version of the awareness day hae day :-) the global organization HAEi and the Spanish HAE organization AEDAF [...]

10Mar, 2016

Pharming’s Report on Preliminary Financial Results 2015

In the financial report for the full year that ended 31 December 2015 Sijmen de Vries, CEO and Chairman of the [...]

9Mar, 2016

Arrowhead Presents Preclinical Data on Possible New HAE Medication

Arrowhead Research Corporation, a biopharmaceutical company developing targeted RNAi therapeutics, has presented additional preclinical data suggesting that ARC-F12, an RNAi therapeutic [...]

9Mar, 2016

Alnylam announcing addition of therapeutic for HAE treatment

Alnylam Pharmaceuticals, Inc. is announcing the addition of a new program to the company’s genetic medicines pipeline, ALN-F12, an investigational [...]

4Mar, 2016

Queen Letizia Accepts Presidency of Honor for HAE Global Conference

It is with the greatest of pleasure that HAEi and AEDAF have received confirmation from the Royal Household of His [...]

4Mar, 2016

Avalanche Biotechnologies Reports Fourth Quarter and Fiscal 2015 Financial Results

Avalanche Biotechnologies, Inc., a gene therapy company committed to discovering and developing novel medicines for patients suffering from chronic or [...]

2Mar, 2016

HAE organization established in Serbia

Over the last few days 21 out of 33 known Serbian HAE families have attended the founding assembly of HAE [...]

1Mar, 2016

More than 400 signed up for HAE Global Conference

We are more than two months away from the opening of the HAE Global Conference 2016 but already we have [...]

1Mar, 2016

Pharming Supports Rare Disease Day

Pharming Group N.V. reiterates it commitment to the treatment of rare diseases on Rare Disease Day 2016, an annual global [...]

27Feb, 2016

Committee for Medicinal Products for Human Use issues positive opinion to European Commission

Following evaluation of a dossier submitted by Pharming Group N.V. last year, the Committee for Medicinal Products for Human Use (CHMP) [...]

24Feb, 2016

BioCryst Reports Full Year 2015 Financial Results

BioCryst Pharmaceuticals, Inc. has announced financial results for the fourth quarter and full year ended December 31, 2015. "In 2015, we gained clarity [...]

12Feb, 2016

Shire announces full year 2015 results

As Shire announces unaudited results for the year to 31 December 2015, Flemming Ornskov, M.D. CEO, comments: "In 2015, we significantly [...]

11Feb, 2016

Zika Virus and Plasma Protein Therapies

Recent scientific and public press reports have heightened awareness of the Zika virus and its emergence in the Americas. The [...]

10Feb, 2016

Shire Marks Anniversary with Global Childrens’ Program

To mark the company’s founding 30 years ago this year, Shire plc is launching a new initiative designed to positively affect [...]

9Feb, 2016

Pharming And Cytobioteck Announce Extension Of Distribution Agreement For Ruconest

Pharming Group N.V. has extended the exclusive distribution agreement with Cytobioteck S.A.S., a privately owned Bogota, Colombia based specialty healthcare [...]

8Feb, 2016

BioCryst Announces Results From OPuS-2

BioCryst Pharmaceuticals, Inc. has announced results from OPuS-2 (Oral ProphylaxiS-2), a clinical trial of avoralstat administered three times daily as a liquid-filled soft gel formulation [...]

2Feb, 2016

Avalanche and Annapurna Announce Proposed Merger

Avalanche Biotechnologies, Inc. and Annapurna Therapeutics SAS have entered into a definitive agreement providing for the acquisition of all outstanding [...]

26Jan, 2016

Come join us in Spain

As you might have noticed in previous information from HAEi, we will be walking part of the pilgrimage route El [...]

24Jan, 2016

Shire Completes Acquisition of Dyax

Shire plc has completed its acquisition of Dyax Corp. in an all-cash transaction. Shire’s CEO, Flemming Ornskov, MD, MPH, commented: “We [...]

12Jan, 2016

Shire to combine with Baxalta, creating the global leader in rare diseases

The boards of directors of Shire plc and Baxalta Incorporated have reached an agreement under which Shire will combine with [...]

6Jan, 2016

Completion Of Patient Enrolment In Clinical Study Of Ruconest

Pharming Group N.V. has completed the patient enrolment in the Phase 2 clinical study of Ruconest, (recombinant C1 esterase Inhibitor, 50 IU/kg), [...]

21Dec, 2015

South Korean Marketing Authorization For Ruconest

Pharming Group N.V.  and it's Seoul based South Korean specialty pharma partner Hyupjin have received the marketing authorization for Ruconest in South [...]

15Dec, 2015

Shire goes to Phase 3 in 2016

In 2016, the Shire plc pipeline will include more than 10 programs in Phase 3 trials and compounds already under regulatory review. Shire’s [...]

10Dec, 2015

CSL Behring Expands Operations to Russia

CSL Behring is opening operations in Russia in order to provide more patients with greater access to treatment. This is particularly significant [...]

7Dec, 2015

EMA Reaffirms Important Health Benefits And Safety Of Ruconest

The European Medicines Agency (EMA) has recently renewed the marketing authorization for Ruconest for an unlimited period. Ruconest, the first and only [...]

26Nov, 2015

Newsletter #7/2015 out now

We are excited to inform you that the seventh edition of the 2015 newsletter is out today. Again, the newsletter offers our [...]

10Nov, 2015

Dyax Corp. presents clinical data from DX-2930 Phase 1b study

Dyax Corp. presented two oral presentations describing clinical data from its DX-2930 Phase 1b study at the American College of [...]

8Nov, 2015

BioCryst Reports Third Quarter 2015 Financial Results

  At the presentation of the BioCryst Pharmaceuticals, Inc. financial results for the third quarter of 2015 Jon P. Stonehouse, [...]

3Nov, 2015

Shire to acquire Dyax, extending industry-leading HAE portfolio

Shire will acquire Dyax Corp., the publicly traded biotechnology company primarily focused on the development of plasma kallikrein (pKal) inhibitors for [...]

28Oct, 2015

More patients are using Ruconest®

Pharming Group N.V. has presented its unaudited financial report for the first nine months ended 30 September 2015. CEO Sijmen De [...]

28Oct, 2015

Newsletter #6/2015 out now

We are excited to inform you that the sixth edition of the 2015 newsletter is out today. Again, the newsletter offers our [...]

16Oct, 2015

Cinryze Receives FDA Fast Track Designation

The United States Food and Drug Administration (FDA) has granted Fast Track designation for the investigation of Cinryze® (C1 esterase inhibitor [human]) [...]

9Oct, 2015

Biocryst Announces Successful Phase 1 Clinical Trial Of BCX7353

BioCryst Pharmaceuticals, Inc. has announced that the randomized, placebo-controlled, Phase 1 clinical trial of orally-administered BCX7353 in healthy volunteers successfully [...]

9Oct, 2015

BioCryst Announces Completion of Patient Enrollment

BioCryst Pharmaceuticals, Inc. has completed enrollment in OPuS-2 (Oral ProphylaxiS-2), a blinded, randomized, placebo-controlled clinical trial of orally-administered avoralstat in patients with HAE. OPuS-2 is [...]

8Oct, 2015

FDA Grants Ruconest Twelve-Year Reference Product Exclusivity

The U.S. Food and Drug Administration (FDA) has granted 12 years of exclusivity to Ruconest® (C1 esterase inhibitor [recombinant]) 50 [...]

1Oct, 2015

Dyax Corp. Receives Positive Opinion in the European Union

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending DX-2930 for designation as an [...]

30Sep, 2015

Sanj K. Patel Appointed to BioCryst’s Board of Directors

BioCryst Pharmaceuticals, Inc. has elected Sanj K. Patel to the Company's Board of Directors. Mr. Patel has a proven track record of successfully developing [...]

16Sep, 2015

Rare disorders process yields first funding decision

People with HAE are the first to benefit from the increased competition for rare disorders medicines promoted by Pharmac, the [...]

10Sep, 2015

Newsletter #5/2015 out now

We are excited to inform you that the fifth edition of the 2015 newsletter is out today. The newsletter comes in a [...]

25Aug, 2015

Shire to attain enhanced Cinryze manufacturing flexibility and capacity

Shire plc has entered into an agreement with Sanquin Blood Supply, the manufacturer of Cinryze® (C1 esterase inhibitor [human]), providing Shire [...]

14Aug, 2015

Prometic reports its second quarter 2015 highlights and financial results

From the Prometic report on the company's second quarter 2015 highlights and financial results: "With our cash position combined with proceeds [...]

13Aug, 2015

Dyax provides update on DX-2930 clinical and commercial supply initiatives

Dyax Corp. has provided an update regarding its ongoing manufacturing initiatives for DX-2930. Discovered by Dyax, DX-2930 is an investigational fully [...]

7Aug, 2015

BioCryst reports second quarter 2015 financial results

BioCryst Pharmaceuticals, Inc. has announced financial results for the second quarter ended 30 June 2015. “During the second quarter, we [...]

1Aug, 2015

Dyax Corp. Announces Second Quarter 2015 Financial Results

Dyax Corp. has announced financial results for the second quarter ended June 30, 2015. Recent highlights include: Receipt of Breakthrough Therapy [...]

30Jul, 2015

Pharming reports on financial results first half year 2015

  Biotech company Pharming Group N.V. has published its (unaudited) financial report for the six months ended 30 June 2015. From [...]

21Jul, 2015

Unique patient organization-driven program

The global collaboration between Clinigen Group plc and Pharming Group N.V. to provide access to Pharming’s Ruconest (conestat alfa) is [...]

21Jul, 2015

HAEi Global Access Program for RUCONEST® Now Live

Unique patient organization-driven program is enabling people with HAE who cannot access effective treatments to receive medication for the first [...]

10Jul, 2015

Newsletter #4/2015 out now

We are excited to inform you that the fourth edition of the 2015 newsletter is out today. Feel free to download it here. [...]

7Jul, 2015

Dyax Corp. Receives FDA Breakthrough Therapy Designation

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigation of DX-2930 for HAE. Dyax Corp. is [...]

11Jun, 2015

Shire appoints Olivier Bohuon to its Board of Directors

Shire plc has appointmented Olivier Bohuon to the Board of Directors as a Non-Executive Director. Olivier will also be a [...]

8Jun, 2015

Dyax Corp. Presents Phase 1b DX-2930 Data

Clinical data from Dyax Corp.'s DX-2930 Phase 1b study was selected as a late-breaking abstract and presented on June 7, 2015 at [...]

1Jun, 2015

Pharming Announces Interim Results From The Ongoing Phase II Pediatric Clinical Trial Of Ruconest

Pharming Group NV presented new results, supporting its EMA and FDA approved HAE therapy RUCONEST®, at the 9th C1- Inhibitor [...]

25May, 2015

Pharming And Cytobioteck Announce Distribution Agreement For Ruconest

Pharming Group NV has entered into an exclusive distribution agreement with Cytobioteck S.A.S., a privately owned Bogota, Colombia based specialty [...]

19May, 2015

HAEi around the globe

HAEi is a global network organization dedicated to raising awareness of C1 inhibitor deficiencies around the world. Our National Member [...]

18May, 2015

Pharming and Clinigen Group Initiate “HAEi GAP” Global Access Program

Pharming Group N.V. and Clinigen Group plc have entered into an international global access collaboration for HAEi. The “HAEi GAP” [...]

16May, 2015

Plans for an exciting HAE Global Conference

Following the very successful HAE conferences in Copenhagen, Denmark in 2012 and Washington D.C., USA in 2014, HAEi is delighted [...]

16May, 2015

Unique HAEi Global Access Program launched

Landmark global program opens access to HAE medicine to rare disease sufferers for the first time Lausanne, Switzerland – [...]

Upcoming Global HAE Events

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Mantra on Russell
Melbourne Australia

Mar 25, 2017 at All Day

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The Grand 14 – Esplanade
Kenner LA United States

Mar 27, 2017 at All Day

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Cinemark Texarkana 14
Texarkana Texas United States

Mar 30, 2017 at All Day

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Recidence Inn
Kingston Ontario Canada

Apr 1, 2017 at All Day

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Hospital Universitario La Paz
Madrid Spain

Apr 1, 2017 at All Day

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Warminster Community Park
Warminster PA United States

Apr 1, 2017 at All Day

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Cinemark Tinseltown
Beaumont TX United States

Apr 3, 2017 at All Day

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Guildhall, Winchester
Winchester United Kingdom

Apr 8, 2017 at All Day

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Hopkins Park
DeKalb IL United States

Apr 9, 2017 at All Day

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UZA
Edegem Belgium

Apr 22, 2017 at All Day

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Parque Central de San Juan
San Juan PR Puerto Rico

Apr 30, 2017 at All Day

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Mantra on Russell
Melbourne Australia

May 13, 2017 at All Day

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Santiago de Compostela Spain

May 13, 2017 at All Day

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Sportsman Lake Park
Cullman AL United States

May 13, 2017 at All Day

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Santiago de Compostela Spain

May 14, 2017 at All Day

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Santiago de Compostela Spain

May 15, 2017 at All Day

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May 16, 2017 at All Day

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Santiago de Compostela Spain

May 16, 2017 at All Day

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Santiago de Compostela Spain

May 17, 2017 at All Day

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Danubius Hotel Margitsziget
Budapest Hungary

May 18, 2017 - May 21, 2017 at 5:00 am - 1:30 pm

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Danada Forest Preserve
Wheaton IL United States

May 20, 2017 at All Day

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Lake Zorinsky Park
Omaha NE United States

Jun 10, 2017 at All Day

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Messukeskus Helsinki
Helsinki Finland

Jun 17, 2017 - Jun 21, 2017 at All Day

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Triemli Spital Zürich
Zürich Switzerland

Jun 24, 2017 at All Day

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- Germany

Aug 3, 2017 - Aug 6, 2017 at All Day

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Miami Whitewater Forest
Harrison OH United States

Sep 9, 2017 at All Day

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Minneapolis Minnesota United States

Sep 15, 2017 - Sep 17, 2017 at All Day

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Normandale Lake Park
Bloomington MN United States

Sep 17, 2017 at All Day

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Roger Williams Park – Carousel Village
Providence RI United States

Oct 7, 2017 at All Day

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Stockholm Sweden

Nov 10, 2017 - Nov 12, 2017 at All Day

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Nov 10, 2017 at All Day

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May 16, 2018 at 12:00 am

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