Nieuws

AVROBIO Announces Clinical and Regulatory Progress for Gaucher Disease Program

15 January 2020

AVROBIO has announced that it has received notice of clearance from the FDA regarding an Investigational New Drug (IND) application for AVR-RD-02, its investigational gene therapy for the treatment of Gaucher disease and has received consent from …

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PR001 Administered to International Type 2 Gaucher Disease Patient Via Compassionate Use Pathway

10 January 2020

Prevail Therapeutics has granted a compassionate use request for the administration of PR001 to a single patient with Type 2 Gaucher disease via a compassionate use pathway, following approval by an international regulatory authority, and the …

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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

8 January 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. About 7 to 10 percent of people with Parkinson’s …

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December 2019 newsletter

17 December 2019

Our latest newsletter includes a report from the 5th International Gaucher conference on bone disease, the executive summary from the African Taskforce and a good news story from Sudan where the IGA was able to get treatment to a young girl.  …

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IGA's membership grows to 55 member associations from 54 countries

12 December 2019

We’re delighted to announce three new patient groups have joined the IGA, bringing our membership up to 55 member associations from 54 countries:

• Colombian Association of Patients with Lysosomal Storage Diseases
• Tunisian Association …

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November 2019 newsletter

28 November 2019

Meet our new team members and read all the latest news from the IGA including an update from the taskforce to improve the lives of Gaucher patients in Pakistan and reports from recent patient meetings in Albania (pictured below) and Belgium - click …

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IGA appoints a new co-opted board member

30 October 2019

Vesna Aleksovska writes:

"As chair of the IGA, I am delighted to announce that Aviva Rosenberg the IGA representative for the National Gaucher Foundation has agreed to join the IGA Board as our new co-opted member. Aviva is a health care …

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October 2019 newsletter

24 October 2019

We have published our latest newsletter, you can read it here

Children from India receiving ERT

11 October 2019

We have always pledged to help any Gaucher patient that asks us for help wherever they live in the world, therefore advocating for charitable treatment has always been and will be our priority.

We are so pleased to be able to share with you (with …

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IGA advocates to get treatment to a young child in Kenya - letter of thanks

11 October 2019

We received a lovely letter from Hassan Suraw, a father of a Gaucher patient in Kenya:

“ I officially received sad news from Head of Hematology that my daughter is having Gaucher Disease last year 2018, while she was 15 months old, and it …

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