Site Logo

News

New Volunteer opportunity
16/04/2021
We are looking for a volunteer patient /patient representative to consult on home therapy research and analysis
PRESS RELEASE: GARDIAN REGISTRY LAUNCH
14/04/2021
The IGA and Kantar Health announce today the launch of the disease registry GARDIAN
PRESS RELEASE: GARDIAN REGISTRY LAUNCH
13/04/2021
PRESS RELEASE: GARDIAN REGISTRY LAUNCH
Become an IGA Volunteer
17/03/2021
Become an IGA Volunteer
February 2021 newsletter
08/03/2021
February 2021 newsletter
Gaucher disease booklet
22/02/2021
Gaucher disease booklet
PR001 in development by Prevail Therapeutics
04/02/2021
PR001 in development by Prevail Therapeutics
January 2021 newsletter
19/01/2021
January 2021 newsletter
Gene Therapy Focus Group
14/01/2021
Gene Therapy Focus Group
Vaccination program for COVID-19 in patients with Gaucher disease
14/01/2021
Vaccination program for COVID-19 in patients with Gaucher disease
A Charitable Access Program for patients with lysosomal storage disorders in underserved communities worldwide
08/01/2021
A Charitable Access Program for patients with lysosomal storage disorders in underserved communities worldwide
A Charitable Access Program for patients with lysosomal storage disorders in underserved communities worldwide
08/01/2021
A Charitable Access Program for patients with lysosomal storage disorders in underserved communities worldwide
December newsletter and office opening hours
08/12/2020
December newsletter and office opening hours
December newsletter and office opening hours
08/12/2020
December newsletter and office opening hours
WATCH NOW: Digital health technology webinar
11/11/2020
WATCH NOW: Digital health technology webinar
WATCH NOW: Digital health technology webinar
11/11/2020
WATCH NOW: Digital health technology webinar
IGA updates its Strategic Plan
10/11/2020
IGA updates its Strategic Plan
IGA updates its Strategic Plan
10/11/2020
IGA updates its Strategic Plan
WATCH NOW: New born screening webinar
04/11/2020
WATCH NOW: New born screening webinar
WATCH NOW: New born screening webinar
04/11/2020
WATCH NOW: New born screening webinar
IGA is granted Charity status
29/10/2020
IGA is granted Charity status
IGA is granted Charity status
29/10/2020
IGA is granted Charity status
Developing digital endpoints for the Gaucher community
15/10/2020
Developing digital endpoints for the Gaucher community
Developing digital endpoints for the Gaucher community
15/10/2020
Developing digital endpoints for the Gaucher community
AVAILABLE TO WATCH NOW: AAV-based gene therapy and Prevail Therapeutic's approach for neuronopathic Gaucher diseases & Parkinson's disease with a GBA mutation
15/10/2020
AVAILABLE TO WATCH NOW: AAV-based gene therapy and Prevail Therapeutic's approach for neuronopathic Gaucher diseases & Parkinson's disease with a GBA mutation
AVAILABLE TO WATCH NOW: AAV-based gene therapy and Prevail Therapeutic's approach for neuronopathic Gaucher diseases & Parkinson's disease with a GBA mutation
15/10/2020
AVAILABLE TO WATCH NOW: AAV-based gene therapy and Prevail Therapeutic's approach for neuronopathic Gaucher diseases & Parkinson's disease with a GBA mutation
Encouraging the voice of the patient in a new trial looking at in utero treatment for nGD at University of California San Francisco
13/10/2020
Encouraging the voice of the patient in a new trial looking at in utero treatment for nGD at University of California San Francisco
Encouraging the voice of the patient in a new trial looking at in utero treatment for nGD at University of California San Francisco
13/10/2020
Encouraging the voice of the patient in a new trial looking at in utero treatment for nGD at University of California San Francisco
AVAILABLE TO WATCH NOW: Ex vivo gene therapy for Type 1 Gaucher disease
05/10/2020
AVAILABLE TO WATCH NOW: Ex vivo gene therapy for Type 1 Gaucher disease
AVAILABLE TO WATCH NOW: Ex vivo gene therapy for Type 1 Gaucher disease
05/10/2020
AVAILABLE TO WATCH NOW: Ex vivo gene therapy for Type 1 Gaucher disease
AVAILABLE TO WATCH NOW: IGD Home Therapy webinar
05/10/2020
AVAILABLE TO WATCH NOW: IGD Home Therapy webinar
AVAILABLE TO WATCH NOW: IGD Home Therapy webinar
05/10/2020
AVAILABLE TO WATCH NOW: IGD Home Therapy webinar
MEMBERS REGISTER NOW for the EWGGD ECongress on 12 & 13 October
02/10/2020
MEMBERS REGISTER NOW for the EWGGD ECongress on 12 & 13 October
MEMBERS REGISTER NOW for the EWGGD ECongress on 12 & 13 October
02/10/2020
MEMBERS REGISTER NOW for the EWGGD ECongress on 12 & 13 October
AVAILABLE TO WATCH NOW - Educational webinar: EWGGD GD1 guidelines
17/09/2020
AVAILABLE TO WATCH NOW - Educational webinar: EWGGD GD1 guidelines
AVAILABLE TO WATCH NOW - Educational webinar: EWGGD GD1 guidelines
17/09/2020
AVAILABLE TO WATCH NOW - Educational webinar: EWGGD GD1 guidelines
AVAILABLE TO WATCH NOW - Educational webinar: Gaucher & Parkinson's disease
17/09/2020
AVAILABLE TO WATCH NOW - Educational webinar: Gaucher & Parkinson's disease
AVAILABLE TO WATCH NOW - Educational webinar: Gaucher & Parkinson's disease
17/09/2020
AVAILABLE TO WATCH NOW - Educational webinar: Gaucher & Parkinson's disease
A letter from Anne-Grethe Lauridsen
08/09/2020
A letter from Anne-Grethe Lauridsen
A letter from Anne-Grethe Lauridsen
08/09/2020
A letter from Anne-Grethe Lauridsen
Online events - Autumn programme
06/08/2020
Online events - Autumn programme
Online events - Autumn programme
06/08/2020
Online events - Autumn programme
Educational webinars
31/07/2020
Educational webinars
Educational webinars
31/07/2020
Educational webinars
Home therapy with enzyme replacement therapy for Gaucher disease: a short statement from the European Working Group on Gaucher Disease (EWGGD)
30/07/2020
Home therapy with enzyme replacement therapy for Gaucher disease: a short statement from the European Working Group on Gaucher Disease (EWGGD)
Home therapy with enzyme replacement therapy for Gaucher disease: a short statement from the European Working Group on Gaucher Disease (EWGGD)
30/07/2020
Home therapy with enzyme replacement therapy for Gaucher disease: a short statement from the European Working Group on Gaucher Disease (EWGGD)
IGD Home Therapy Photo Competition
22/07/2020
IGD Home Therapy Photo Competition
AVROBIO Announces New Patients Dosed in Gaucher Disease and Cystinosis Clinical Trials
06/07/2020
AVROBIO Announces New Patients Dosed in Gaucher Disease and Cystinosis Clinical Trials
Orphazyme phase 2 study of arimoclomol in Gaucher disease
24/06/2020
Orphazyme phase 2 study of arimoclomol in Gaucher disease
IGA and CENTOGENE Team Up to Ensure myLSDapp Provides the Highest Level of Patient Data Protection
15/06/2020
IGA and CENTOGENE Team Up to Ensure myLSDapp Provides the Highest Level of Patient Data Protection
COVID-19: IGA and European GD Network Gaucher Research Survey
29/05/2020
COVID-19: IGA and European GD Network Gaucher Research Survey
Minutes from IGA's Biennial General Meeting
29/05/2020
Minutes from IGA's Biennial General Meeting
The IGA initiates a global discussion on home therapy for Gaucher patients
14/05/2020
The IGA initiates a global discussion on home therapy for Gaucher patients
AVROBIO to Collaborate with Saladax Biomedical
11/05/2020
AVROBIO to Collaborate with Saladax Biomedical
COVID19 & home treatment
28/04/2020
COVID19 & home treatment
Notes from the EWGGD webinar on 1 April
14/04/2020
Notes from the EWGGD webinar on 1 April
Results of the IGA's Board Election
14/04/2020
Results of the IGA's Board Election
The IGA has co-authored 'The Definition of Neuronopathic Gaucher disease'
07/04/2020
The IGA has co-authored 'The Definition of Neuronopathic Gaucher disease'
IGA co-authors a scientific paper titled 'The road to biosimilars in rare diseases – ongoing lessons from Gaucher disease
06/04/2020
IGA co-authors a scientific paper titled 'The road to biosimilars in rare diseases – ongoing lessons from Gaucher disease
COVID-19: A statement from the EWGGD
31/03/2020
COVID-19: A statement from the EWGGD
Covid-19 - keeping in touch
30/03/2020
Covid-19 - keeping in touch
An update from the ORPHAZYME Advocacy Team
30/03/2020
An update from the ORPHAZYME Advocacy Team
An update from the TAKEDA Advocacy team
30/03/2020
An update from the TAKEDA Advocacy team
An update from the AVROBIO advocacy team
25/03/2020
An update from the AVROBIO advocacy team
IGA's membership grows to 57 member associations
23/03/2020
IGA's membership grows to 57 member associations
COVID-19 : Supporting our members
20/03/2020
COVID-19 : Supporting our members
March 2020 newsletter
18/03/2020
March 2020 newsletter
A message regarding the COVID-19 pandemic
18/03/2020
A message regarding the COVID-19 pandemic
IGA response to COVID-19: attending meetings
16/03/2020
IGA response to COVID-19: attending meetings
Good news sharing - charitable access
13/03/2020
Good news sharing - charitable access
IGA neuronopathic Gaucher Disease (nGD) Global Registry: patient community phase I final report
12/03/2020
IGA neuronopathic Gaucher Disease (nGD) Global Registry: patient community phase I final report
Postponement of IGA Biennial Meeting in response to concerns over COVID-19
10/03/2020
Postponement of IGA Biennial Meeting in response to concerns over COVID-19
Would you like to join the IGA's board of directors?
25/02/2020
Would you like to join the IGA's board of directors?
Kantar partnership announcement: Facebook Live session
05/02/2020
Kantar partnership announcement: Facebook Live session
14th European Working Group on Gaucher Disease Congress
04/02/2020
14th European Working Group on Gaucher Disease Congress
International Gaucher Alliance and Kantar Partnership announcement
03/02/2020
International Gaucher Alliance and Kantar Partnership announcement
IGA's Membership Grows to 56 Member Associations From 54 Countries
23/01/2020
IGA's Membership Grows to 56 Member Associations From 54 Countries
AVROBIO Announces Clinical and Regulatory Progress for Gaucher Disease Program
15/01/2020
AVROBIO Announces Clinical and Regulatory Progress for Gaucher Disease Program
PR001 Administered to International Type 2 Gaucher Disease Patient Via Compassionate Use Pathway
10/01/2020
PR001 Administered to International Type 2 Gaucher Disease Patient Via Compassionate Use Pathway
Prevail Therapeutics is recruiting patients for PROPEL, a clinical trial testing an investigational gene therapy called PR001 to treat Parkinson’s disease in people with GBA mutations
08/01/2020
Prevail Therapeutics is recruiting patients for PROPEL, a clinical trial testing an investigational gene therapy called PR001 to treat Parkinson’s disease in people with GBA mutations
December 2019 newsletter
17/12/2019
December 2019 newsletter
IGA's membership grows to 55 member associations from 54 countries
12/12/2019
IGA's membership grows to 55 member associations from 54 countries
November 2019 newsletter
28/11/2019
November 2019 newsletter
IGA appoints a new co-opted board member
30/10/2019
IGA appoints a new co-opted board member
October 2019 newsletter
24/10/2019
October 2019 newsletter
Children from India receiving ERT
11/10/2019
Children from India receiving ERT
IGA advocates to get treatment to a young child in Kenya - letter of thanks
10/10/2019
IGA advocates to get treatment to a young child in Kenya - letter of thanks
Understanding the impact of being diagnosed with a comorbidity
10/10/2019
Understanding the impact of being diagnosed with a comorbidity
Happy International Gaucher Day
01/10/2019
Happy International Gaucher Day
September 2019 newsletter
26/09/2019
September 2019 newsletter
Regional Managers visit BP Koirala Hospital in Dharan, Nepal
11/09/2019
Regional Managers visit BP Koirala Hospital in Dharan, Nepal
Over 53 paediatricians attend event hosted by IGA in Kathmandu
10/09/2019
Over 53 paediatricians attend event hosted by IGA in Kathmandu
Regional Managers organise LSD patient meeting in Kathmandu
09/09/2019
Regional Managers organise LSD patient meeting in Kathmandu
Regional Managers organise medical programme in Nepal
06/09/2019
Regional Managers organise medical programme in Nepal
Orphazyme complete enrolment in Phase II clinical trial
30/08/2019
Orphazyme complete enrolment in Phase II clinical trial
August 2019 newsletter
29/08/2019
August 2019 newsletter
IGA expands its global reach to Central America
07/08/2019
IGA expands its global reach to Central America
Sanofi Genzyme provide ERT to three Sudanese children through their Charitable Access Programme
06/08/2019
Sanofi Genzyme provide ERT to three Sudanese children through their Charitable Access Programme
Six year old Tanzanian child gets treatment
06/08/2019
Six year old Tanzanian child gets treatment
New Croatian Gaucher Association joins the IGA
22/07/2019
New Croatian Gaucher Association joins the IGA
IGD Press Release
17/07/2019
IGD Press Release
nGD poster presentation at the EWGGD
08/07/2019
nGD poster presentation at the EWGGD
Jeremy Manuel is the IGA's rare star for IGD2019
04/07/2019
Jeremy Manuel is the IGA's rare star for IGD2019
July Newsletter (IGD)
04/07/2019
July Newsletter (IGD)
Brazil to join the IGA
26/06/2019
Brazil to join the IGA
June newsletter
24/06/2019
June newsletter
ORDER YOUR #IGD2019 TATTOOS - MEMBERS ONLY
04/06/2019
ORDER YOUR #IGD2019 TATTOOS - MEMBERS ONLY
NOMINATE YOUR RARE STAR FOR #IGD2019
04/06/2019
NOMINATE YOUR RARE STAR FOR #IGD2019
International Gaucher Day 2019 #IGD2019
31/05/2019
International Gaucher Day 2019 #IGD2019
May newsletter
28/05/2019
May newsletter
April newsletter
25/04/2019
April newsletter
AVROBIO explore gene therapy as a potential treatment for Gaucher disease
10/04/2019
AVROBIO explore gene therapy as a potential treatment for Gaucher disease
IGA appoints a new co-opted member to its board
09/04/2019
IGA appoints a new co-opted member to its board
March newsletter
26/03/2019
March newsletter
Election of new Chair and Vice-Chair
06/03/2019
Election of new Chair and Vice-Chair
February newsletter
28/02/2019
February newsletter
Pascal Niemeyer resigns as IGA Chairman and Director
13/02/2019
Pascal Niemeyer resigns as IGA Chairman and Director
Australia & New Zealand to join the IGA
12/02/2019
Australia & New Zealand to join the IGA
Looking for Volunteers
22/01/2019
Looking for Volunteers
January 2019 newsletter
22/01/2019
January 2019 newsletter
Global Patient Registry for nGD
14/01/2019
Global Patient Registry for nGD
December newsletter
18/12/2018
December newsletter
Mongolia to join the IGA
10/12/2018
Mongolia to join the IGA
November newsletter
30/11/2018
November newsletter
Minutes from the EGA's Biennial Members Meeting
21/11/2018
Minutes from the EGA's Biennial Members Meeting
October newsletter
01/11/2018
October newsletter
EWGGD 2019 'Save the Date'
24/10/2018
EWGGD 2019 'Save the Date'
The EGA becomes the IGA
14/10/2018
The EGA becomes the IGA
#IGD2018 #rarestars videos
02/10/2018
#IGD2018 #rarestars videos
September newsletter
24/09/2018
September newsletter
Turkey and Kazakhstan to join the EGA
11/09/2018
Turkey and Kazakhstan to join the EGA
August Newsletter
12/08/2018
August Newsletter
Biennial Members Meeting – 15 & 16 October 2018
04/07/2018
Biennial Members Meeting – 15 & 16 October 2018
Results of EGA 2018/2020 Election
27/06/2018
Results of EGA 2018/2020 Election
Morocco to join the EGA
21/06/2018
Morocco to join the EGA
GDPR Changes. Tell us how you want to be contacted.
30/04/2018
GDPR Changes. Tell us how you want to be contacted.
Pakistan to join the EGA
22/04/2018
Pakistan to join the EGA
EGA Board Membership Opportunities
10/04/2018
EGA Board Membership Opportunities
Advocacy training grant
10/04/2018
Advocacy training grant
Important Changes to Data Protection.
07/02/2018
Important Changes to Data Protection.
Rare Disease Day Video 2018
30/01/2018
Rare Disease Day Video 2018
Rare Disease Day 2018 28 February
15/01/2018
Rare Disease Day 2018 28 February
Seasons Greetings and Office Opening Hours
19/12/2017
Seasons Greetings and Office Opening Hours
International Gaucher Day 2017. A great success.
03/10/2017
International Gaucher Day 2017. A great success.
A further Inspiring #IGD2017 tale for you to enjoy.
27/09/2017
A further Inspiring #IGD2017 tale for you to enjoy.
3rd IGD 2017 story for you to enjoy!
22/09/2017
3rd IGD 2017 story for you to enjoy!
2nd Inspiring story for IGD 2017 published
19/09/2017
2nd Inspiring story for IGD 2017 published
IGD2017
12/09/2017
IGD2017
Go with gaucher
20/07/2017
Go with gaucher
Clinical Trial for Gaucher Disease Type 3
22/02/2017
Clinical Trial for Gaucher Disease Type 3
Seasons Greetings/Office opening hours
16/12/2016
Seasons Greetings/Office opening hours
Botswana to join the EGA
29/11/2016
Botswana to join the EGA
Go With Gaucher, taking forward the next generation: how to involve young people to create a new generation of patient advocates
03/11/2016
Go With Gaucher, taking forward the next generation: how to involve young people to create a new generation of patient advocates
Your opinion about an app to report side-effects and receive safety information
07/09/2016
Your opinion about an app to report side-effects and receive safety information
A new Korean ERT 'Abcertin' - Important Information for doctors treating Gaucher patients
30/08/2016
A new Korean ERT 'Abcertin' - Important Information for doctors treating Gaucher patients
Spotlight on Gaucher 2016 Campaign
28/08/2016
Spotlight on Gaucher 2016 Campaign
Change to the constitution: Members Vote paves the way to the EGA becoming global
18/07/2016
Change to the constitution: Members Vote paves the way to the EGA becoming global
EGA announces new chairman
18/07/2016
EGA announces new chairman
Dr Roscoe Brady
20/06/2016
Dr Roscoe Brady
Guatemala and Japan to join the EGA
13/04/2016
Guatemala and Japan to join the EGA
EGA GOES TO SOUTH AFRICA
13/04/2016
EGA GOES TO SOUTH AFRICA
Launch of the Official Website of the 12th EWGGD Meeting In Zaragoza, Spain
17/03/2016
Launch of the Official Website of the 12th EWGGD Meeting In Zaragoza, Spain
Shire marks 30th Anniversary with Innovative Global Program to benefit children with Rare Diseases
17/02/2016
Shire marks 30th Anniversary with Innovative Global Program to benefit children with Rare Diseases
China to join the EGA
10/02/2016
China to join the EGA
The Patient Group Handbook: A Practical Guide for Research and Drug Development
11/01/2016
The Patient Group Handbook: A Practical Guide for Research and Drug Development
The Psychosocial and Neurocognitive Impacts of Gaucher Disease, Type 1 on Pediatric and Young Adult Patients
23/11/2015
The Psychosocial and Neurocognitive Impacts of Gaucher Disease, Type 1 on Pediatric and Young Adult Patients
Launch of the 'Spotlight on Gaucher Film Contest'
15/07/2015
Launch of the 'Spotlight on Gaucher Film Contest'
UK Gauchers Association Granted a Positive Opinion For Orphan Designation For Gene Therapy For Gaucher Disease
27/02/2015
UK Gauchers Association Granted a Positive Opinion For Orphan Designation For Gene Therapy For Gaucher Disease
Watch the Official Rare Disease Day 2015 Video!
17/02/2015
Watch the Official Rare Disease Day 2015 Video!
European Commission Grants Marketing Authorization for Cerdelga (eliglustat), Genzyme`s oral therapy for Gaucher Disease Type 1
23/01/2015
European Commission Grants Marketing Authorization for Cerdelga (eliglustat), Genzyme`s oral therapy for Gaucher Disease Type 1
European CHMP Adopts Positive Opinion for Genzyme's Cerdelga (eliglustat) Capsules
23/11/2014
European CHMP Adopts Positive Opinion for Genzyme's Cerdelga (eliglustat) Capsules
Genzyme Announces Launch of `Expression of Hope III` Program to Build Awareness of Lysosomal Storage Disorders Through Works of Art
16/09/2014
Genzyme Announces Launch of `Expression of Hope III` Program to Build Awareness of Lysosomal Storage Disorders Through Works of Art
FDA Approval of Pediatric Indication for ELELYSO (taliglucerase alfa)
28/08/2014
FDA Approval of Pediatric Indication for ELELYSO (taliglucerase alfa)
FDA Approves Genzyme's Cerdelga (eliglustat) Capsules
20/08/2014
FDA Approves Genzyme's Cerdelga (eliglustat) Capsules
Joint proposal to facilitate clinical investigation of new medicines for Gaucher disease in children
17/05/2014
Joint proposal to facilitate clinical investigation of new medicines for Gaucher disease in children
FDA Grants Priority Review for Cerdelga (Eliglustat)
16/12/2013
FDA Grants Priority Review for Cerdelga (Eliglustat)
Ega becomes full member of EURORDIS
28/11/2013
Ega becomes full member of EURORDIS
Luxembourg to join the EGA
27/11/2013
Luxembourg to join the EGA
Moldova to join the EGA as 40th member
19/09/2013
Moldova to join the EGA as 40th member
6th nGD Family Conference, 8-10.11.2013 in Reading, UK.
02/07/2013
6th nGD Family Conference, 8-10.11.2013 in Reading, UK.
Croatia to join the EGA
18/06/2013
Croatia to join the EGA
Paraguay to join the EGA
11/06/2013
Paraguay to join the EGA
Report from regional patient meeting in Sarajevo, Bosnia & Herzegovina
20/04/2013
Report from regional patient meeting in Sarajevo, Bosnia & Herzegovina
Canada to join the EGA
26/03/2013
Canada to join the EGA
Alert for Gaucher Patients and Families regarding Zavesca Getz Product
14/12/2012
Alert for Gaucher Patients and Families regarding Zavesca Getz Product
EC denies Taliglucerase (Elelyso) market approval for Type 1 GD
06/11/2012
EC denies Taliglucerase (Elelyso) market approval for Type 1 GD
Genzyme Phase 3 Study of Eliglustat Meets Primary Endpoint
18/10/2012
Genzyme Phase 3 Study of Eliglustat Meets Primary Endpoint
Market approval of Elelyso by the Israeli Ministry of Health
03/10/2012
Market approval of Elelyso by the Israeli Ministry of Health
India to join the EGA
27/09/2012
India to join the EGA
Mexico to join the EGA
24/05/2012
Mexico to join the EGA
Bosnia & Herzegovina to join EGA
03/05/2012
Bosnia & Herzegovina to join EGA
FDA approved Elelyso (taliglucerase alfa)
01/05/2012
FDA approved Elelyso (taliglucerase alfa)
Macedonia and Switzerland to join EGA
19/04/2012
Macedonia and Switzerland to join EGA
Humanitarian Aid (March 2012)
26/03/2012
Humanitarian Aid (March 2012)
Shire Announces European Approval of Manufacturing Facility for VPRIV
22/02/2012
Shire Announces European Approval of Manufacturing Facility for VPRIV
Press release - Genzyme: Four-Year Data from Phase 2 Trial ...
09/02/2012
Press release - Genzyme: Four-Year Data from Phase 2 Trial ...
Press release: Protalix Announces New Clinical Data on Taliglucerase Alfa
09/02/2012
Press release: Protalix Announces New Clinical Data on Taliglucerase Alfa
EGA is now officially eligible to be involved in the acitivities of the EMA
21/12/2011
EGA is now officially eligible to be involved in the acitivities of the EMA
1st Regional Patient Meeting in Slovenia
21/11/2011
1st Regional Patient Meeting in Slovenia
New EGA Website launched
01/11/2011
New EGA Website launched
Cerezyme Emergency Treatment Program (CETP)
04/01/2011
Cerezyme Emergency Treatment Program (CETP)
Achive