California Rare Disease Access Coalition

08/10/2026

Access to plasma-derived medicines starts with a strong donation system. We urge the CA Senate Appropriations Committee to pass , a key step toward modernizing plasma donation center regulations and increasing access to these life-saving medicines.

07/29/2026

Social determinants of health account for up to 80% of health outcomes in the U.S. Integrating interventions into health care can improve outcomes, reduce avoidable care, and advance more equitable, whole-person care.
Read more here: https://tinyurl.com/3xwjdf7z

07/28/2026

A recent study found that children with hemophilia A have distinct immune and inflammatory reactions, suggesting that the type of preventive treatment they receive may influence their responses.
Read more here:

Hemophilia A treatments were linked to different immune patterns in children in a small study of Hemlibra and factor VIII therapy.

07/24/2026

Copay accumulators let insurers & keep your copay assistance without counting toward your deductible. When it runs out, you pay again. Proud to join 90+ orgs urging & to enforce EHB protections & ensure . https://bit.ly/4bNEJ7k

07/23/2026

Gene therapy is transforming medicine by targeting disease at its source, offering new hope for patients with Huntington’s disease, sickle cell disease, inherited neurological disorders, and other genetic conditions.
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Find out how gene therapy is changing how we treat disease with groundbreaking research and innovative technologies leading the way.

07/22/2026

We are proud to join 90+ patient and provider groups in urging to strengthen prescription drug coverage and enforce Essential Health Benefits (EHB) protections. Read the letter here: https://bit.ly/4bNEJ7k

07/22/2026

Join NORD for two free, virtual Living Rare, Living Stronger® webinars designed for patients, caregivers and families navigating a rare disease.

From 3-4 p.m. ET on July 30, learn about common insurance barriers and practical tips to overcome them. RSVP: https://bit.ly/4pqrkIc

From 3-4 p.m. ET on Aug. 27, explore new approaches to improving your rare disease care. RSVP: https://bit.ly/4vZmIei

Both sessions include live Q&A and allow you to submit questions in advance. They will both be recorded for those who cannot tune in live!

07/21/2026

A new rare disease initiative aims to strengthen communities and clinical studies, helping patients, physicians, and scientists work together to advance research and identify new treatment opportunities.
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Chan Zuckerberg Initiative's Biohub will offer new grants to the rare disease community while expanding its AI drug repurposing partnership with Every Cure.

07/16/2026

A new gene therapy for rare immune disorder shows promise in early pre-clinical studies for the treatment of CTLA-4 insufficiency.
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The development of a pioneering gene therapy to treat a devastating, rare immune disorder led by UCL scientists is set to move into its next phase following promising early pre-clinical studies.

07/11/2026

Another advocacy opportunity for the rare disease community!