News

February 13, 2020February 14, 2020

Sanofi’s Next-generation ERT Halts Progression of Pompe Disease in Patients, Interim Trial Data Show

News

Sanofi Genzyme’s next-generation enzyme replacement therapy (ERT), avalglucosidase alfa, safely halts disease progression in people with Pompe disease, according to interim data from two Phase 2 clinical trials. The ... Read more

February 12, 2020February 12, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 6, 2020February 6, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

News

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

February 6, 2020February 6, 2020

MDA Leads in Efforts to Help Patients With Neuromuscular Diseases – From Care Centers to Research Grants and Summer Camp

News

Expecting to award roughly $18 million this year in grants to support research in neuromuscular diseases, the Muscular Dystrophy Association (MDA) continues to be a leader in efforts to better ... Read more

February 6, 2020February 6, 2020

Gene Therapies, Redosing, and Other Work Underway in Treating Pompe: Talk With MDA

News

Gene therapies — after a tragedy kicked them back to the lab two decades ago — are beginning to come into their own, making possible the progress in gene-targeted ... Read more

January 30, 2020January 30, 2020

AskBio Launches AskFirst Advocacy Program to Educate and Support Gene Therapy Trial Participants

News

Asklepios Biopharmaceutical (AskBio) has introduced a collaborative program designed to educate patients who may benefit from gene therapy, and to support those in clinical trials. Called AskFirst, the program ... Read more

January 23, 2020January 23, 2020

CHOC Children’s Given $8M to Develop Next-Generation Therapies for Pompe Disease

News

The Foundation of Caring has given $8 million to support CHOC Children’s efforts to develop next-generation therapies for Pompe disease. In honor of the gift, the program has been ... Read more

January 16, 2020January 16, 2020

Amicus Completes Patient Enrollment for Phase 3 Trial of AT-GAA; Remains on Track to File BLA in 2021

News

Amicus Therapeutics has completed and exceeded patient enrollment for its Phase 3 PROPEL trial of AT-GAA, its investigational chaperone therapy for the treatment of Pompe disease. The company also ... Read more

January 9, 2020January 9, 2020

The Assistance Fund Now Includes Pompe Disease

News

To help patients and families facing out-of-pocket medical costs, The Assistance Fund (TAF) has opened a new program for eligible individuals living with Pompe disease. The program assists with treatment-related ... Read more

January 2, 2020January 2, 2020

Problems with Diaphragm Evident in Lung Function Tests of LOPD Patients, Study Reports

News

People with late-onset Pompe disease (LOPD) have lower dynamic diaphragm function and altered endurance and strength during inhalation, but they show some compensatory mechanisms to preserve air volume, according to ... Read more