Ever wonder why HDSA and the HD research community is always talking about Enroll-HD? Are you curious about the HD research foundation known as CHDI and what they do? Do you want to know how participants in studies that don’t involve any drugs (known as observational research) are actually speeding up the discovery of new drugs? Watch this presentation from HDSA’s 2019 Convention by Dr. Robert Pacifici, Chief Scientific Officer of CHDI Foundation. HDSA’s Dr. George Yohrling called it “the most important talk of the conference.” It’s a really great way to better understand exactly what HD researchers are learning from the individuals and families who choose to participate in observational research studies, and to find out about ongoing and upcoming studies that you can join to make a difference.
New Patient-Focused Pages On The uniQure Website
UniQURE’s mission is to create novel, single-treatment gene therapies for patients with genetic diseases. This June they released a letter to the community announcing their intention to begin an early safety trial of a Huntington’s disease gene therapy called AMT-130. This week they launched some new pages on their website about Huntington’s disease and gene therapy. Stay tuned as we hope to hear more from UniQURE in the coming months as their clinical trial plans move forward.
Cigna To Cover Gene Therapy For SMA & Rare Eye Disease
Cigna, a major health insurance provider, has announced that it will cover two gene therapies at no cost to patients: Zolgensma, a drug that treats spinal muscular atrophy (SMA) in children under two, and Luxturna, a treatment for Inherited Retinal Disease (IRD). While this news does not immediately impact the HD community, Cigna has set an important precedent that could influence coverage of other gene therapies in the future. UniQURE is poised to begin the first gene therapy trial for Huntington’s disease, and many other companies are working in this space, including Spark Therapeutics, the company that makes Luxturna.
Patient advocacy and care organizations like HDSA work hard to ensure that families with rare disorders will have access to life-changing therapies when they arrive, and we’re excited to hear that a major payer is helping to meet that goal for the SMA and IRD communities.
