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 is intended to keep patients and families apprised of the company’s gene therapy research and potential cures for…
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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 renamed the Foundation of Caring Lysosomal Storage Disorder Program at CHOC Children’s. The comprehensive multidisciplinary program…
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 remains on track to file a biologics license application…
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 copayments, health insurance premiums and incidental medical expenses related to the metabolic disorder that affects roughly one in every…
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 a recent study. These findings, which were achieved using dynamic — rather than the standard, static — lung function…
Patients with Pompe disease also may exhibit motor neuronopathy (motor and nerve deficits due to neuron degeneration) in addition to the classical myopathy (muscle disease) features, a case study suggests. Researchers suggest that future studies also should focus on therapies that target the central nervous system, and that…
The Muscular Dystrophy Association is set to host the first Pompe disease Patient-Focused Drug Development (PFDD) meeting next year to inform therapy developers, regulators, and other stakeholders about the experiences of Pompe disease patients and caregivers, as well as their expectations for future treatments. Patients and family members interested…
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
A glance around the walls of Barry J. Byrne’s office reveals a lot about the pediatric cardiologist who runs the Powell Gene Therapy Center at University of Florida (UF). In one corner is an unusual painting by 9-year-old Will Barkowsky of Jacksonville, Fla. Will, the first boy with…