Rare New England

06/05/2026

Don't forget our caregiver support group meets the 2nd Friday of every month (new date)!
https://www.rarenewengland.org/rare-connections

06/04/2026

"AI can define the population most likely to benefit and shape how outcomes are measured. It can inform trial design in ways that reduce avoidable failure and improve the quality of evidence generated."

The FDA has introduced models intended to accelerate rare disease drug development, but recent reversals of guidance from the agency speak to a lack of clarity in its implementation. AI can help focus this process.

06/03/2026

"Their mother is off to Maryland to go all in against a disease where one loses bone faster than it can grow. It’s a human trial for one at the National Institutes of Health. Neena Nizar is the only person taking part."

Human trials featuring just one participant are rare. But when a condition is so rare it’s impossible to find enough people for a standard trial, it often falls to one person.

06/03/2026

Rare New England Rare Connections Caregiver Support Group 💜

Join us on Friday, June 19 at 6:00 PM ET for our monthly caregiver support group!

This is a welcoming space for rare disease caregivers to connect, share experiences, ask questions, and support one another. With summer break beginning for many families, we'll discuss how the season impacts our routines, caregiving responsibilities, activities, appointments, travel plans, and more.

Whether you're looking for support, resources, or simply a community that understands, we'd love to have you join us.

📅 Friday, June 19
⏰ 6:00 PM ET
💻 Virtual via Zoom

📧 The Zoom link will be emailed to registered attendees the morning of the event.

We look forward to connecting with you and hearing how your family is preparing for summer!

06/02/2026

Rare New England Rare Connections Caregiver Support Group – Special Guest Speaker Event

We're excited to invite you to a special Rare Connections Caregiver Support Group presentation featuring Lauren Masto, M.S., CCC-SLP, a pediatric feeding specialist, speech-language pathologist, and mom of two.

With over 10 years of experience helping children and families navigate feeding challenges, Lauren is passionate about creating positive, pressure-free mealtime experiences that build confidence, reduce stress, and help children develop healthy relationships with food. She has extensive experience working with infants through teens, including children with feeding tubes and complex medical needs, and loves partnering with families to celebrate every milestone—big or small.

As both a feeding specialist and a parent to Brooke (5) and Gavin (3), Lauren brings not only professional expertise but also a personal understanding of the joys and challenges families experience every day.

📅 Monday, June 29
⏰ 6:00–7:00 PM ET
💻 Virtual via Zoom

This special presentation is open to all rare disease families, caregivers, patients, and professionals. Please invite friends and share with anyone who may benefit!

📋 Register here or scan QR code: https://docs.google.com/forms/d/1yepA0Cj96hLxFer9ITT22XXvRDHdHkShEayoMbmL1QA/edit

📧 The Zoom link will be emailed to registered attendees the morning of the event.

We hope you'll join us for this informative discussion on building positive feeding experiences, reducing mealtime stress, and supporting medically complex children and families throughout their feeding journey.

06/02/2026

"the researchers were able to use their ancient ancestor-based protein interactome — essentially a map of protein networks — to identify previously unknown genes associated with disease."

Researchers have identified genes previously unknown to be connected to three rare disorders, a discovery that sheds new light on the genetic causes of human diseases.

06/01/2026

Really interesting article focusing on the Nicolaides-Baraitser syndrome community

Dramatic cuts to science research grants have particularly affected rare disease research. But some of the people most affected, including those with Nicolaides-Baraitser syndrome, are now organizing...

05/30/2026

05/29/2026

/PRNewswire/ -- On Tuesday, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened its biennial Scientific Workshop titled, "Making What...

05/28/2026

Don't forget our patient support group that meets the 1st Tuesday of every month!
https://www.rarenewengland.org/rare-connections