Mary Chapman,  features writer—

Neurogene, a biopharmaceutical company developing therapies for Batten disease, is trying to learn more about the progression of the subtype CLN5 disease from the caregiver perspective. To that end, Neurogene is inviting the caregivers of children who are either living with CLN5, or who died within the…

Families of a child with Batten disease in the U.K. are welcome take part in counseling and other wellness services being offered at no cost by the Maypole Project in a three-year collaboration with the Batten Disease Family Association (BDFA). Services include one-on-one therapy sessions, as well as sessions tailored to couples,…

Editor’s note: This story was updated March 30, 2021 to clarify that Neurogene is hosting videoconferences workshops for families in any country. Neurogene, which is developing treatments for Batten disease and other rare neurological disorders, is inviting families affected by CLN5, a form of late infantile…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

The Batten Disease Family Association (BDFA) is moving forward with a fundraising campaign aimed at developing a United Kingdom study that would use Brineura (cerliponase), an enzyme replacement therapy (ERT), to try to maintain eyesight in children with a type of Batten disease known as…