Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of rare and orphan diseases, today announced that its investigational new drug (IND) application, submitted to U.S. Food and Drug Administration (FDA), is now effective which allows Amicus to begin site initiation and enrollment of a Phase 1/2 study of ATB200 in patients with Pompe disease.

Rare Disease Group Urges Ontario to Include Devastating Neuromuscular Condition in Newborn Screening Program

Rare Disease Group Urges Ontario to Include Devastating Neuromuscular Condition in Newborn Screening Program

TORONTO (Sept. 11, 2015) – The Canadian Association of Pompe (CAP) today submitted a nomination for Pompe disease to be considered for addition in Ontario’s newborn screening panel. In recognition of Newborn Screening Awareness Month, CAP urges Newborn Screening Ontario to expand its program to include this rare neuromuscular condition. Without early diagnosis and treatment, infants with this rare genetic illness are not likely to survive past the age of one.

Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Genzyme Recognizes International Rare Disease Day with Global Education and Fund-Raising Events; Announces Founding Partnership of Give RARE

Genzyme, a Sanofi company, announced today that in support of International Rare Disease Day, the company is hosting several events internationally to help raise awareness of rare diseases as a critical global health issue requiring ongoing research, education and new treatments. Genzyme also announced that it is a founding partner of Give RARE, a rare diseases global giving day.

Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Audentes Therapeutics Joins Global Organizations In Celebrating Rare Disease Day® 2015

SAN FRANCISCO, CA – February 26, 2015 – Audentes Therapeutics, Inc., a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, today announced its support for Rare Disease Day®, to be held on February 28th, 2015. The theme of this year’s Rare Disease Day is Living with a Rare Disease. In honor of the day, Audentes is pleased to announce the launch of a website.

Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

New Brunswick Announces Plan to Treat Patients

The provincial government has established the New Brunswick Drugs for Rare Diseases Plan which will provide assistance to those with certain rare diseases who face high drug costs. New Brunswick will partner with Ontario to deliver the plan using its Drugs for Rare Diseases Framework. The framework was established to assess drugs using the best available evidence and recommend drugs for funding based on clinical criteria.

Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Genzyme Recognizes International Rare Disease Day with Global Awareness Programs and Launch of Fourth Annual Patient Advocacy Leadership (PAL) Awards

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced its support of International Rare Disease Day with a series of initiatives meant to call attention to rare diseases as an important public health issue and to improve rare disease education, research, and treatment.