Stop ALD Foundation

https://discoveries.childrenshospital.org/first-gene-therapy-ald-recipient/?fbclid=IwAR10OI1qQ6Dqq0gjX_Go3h2KnJa9EFgckVAZja2JwfRGn5y7BKSS63mZH3o

Matthew, the ‘wee marvel’: One of the first ALD gene therapy recipients - Boston Children's Answers Read about Matthew Elliott, the first patient in the world to receive gene therapy for adrenoleukodystrophy (ALD).

https://bostonchildrens.zoom.us/webinar/register/WN_r6MF4-MiQy6sGmTngGLsAA

You’re invited to join us in celebrating the 10-year anniversary of the Boston Children's Hospital Gene Therapy Program. After registering, you will receive a confirmation email with the virtual event information. You’re invited to join us in celebrating the 10-year anniversary of the Boston Children's Hospital Gene Therapy Program. You’ll meet members of the Gene Therapy team, hear from some of our gene therapy recipients about their experiences and have an opportunity to ask questions. _________________...

Learn more about next month's ALD Family Weekend March 19-21, 2021!

ALD Family Weekend The ALD Family Weekend is a unique experience that allows families affected by adrenoleukodystrophy to connect in a fun environment - The Painted Turtle Camp. Parents, caregivers are encouraged to attend educational presentations by ALD specialists and physicians who are active in the research and t

ALD Family Weekend is May 1-3 in Lake Hughes, CA at The painted turtle camp. Looking forward to connecting, learning and having fun with the community. ALD Family Weekend

ALD Family Weekend

Important Stanford Medicine survey for caregivers of children with X-ALD to help better inform healthcare professionals. Plz complete by 28 Feb 2020.

X-ALD Caregiver Survey

BE HEARD: Share your story with staff of the U.S. Food and Drug Administration about your experience of living with Childhood Cerebral Adrenoleukodystrophy (CCALD).
FDA’s Patient Affairs Staff (PAS and the National Organization for Rare Disorders, Inc. (NORD) are hosting a “Listening Session” for Childhood Cerebral Adrenoleukodystrophy (CCALD).

This session is a private, confidential conference call where a small, group of people affected by Childhood Cerebral Adrenoleukodystrophy (CCALD) can share first-hand with FDA staff their experiences of living with this rare disease. Participants are selected based upon diversity of gender,
geographic location, disease severity and other factors.
Our organization is involved with the hope to provide information from the patient perspective that
will help FDA staff members better understand the burden of living with this disease, the needs and concerns of patients.
There are a limited number of openings for participating in the session, if you are interested in participating on this call, please let us know by September 30 by filling out this brief survey:

CCALD FDA Rare Disease Listening Session Take this survey powered by surveymonkey.com. Create your own surveys for free.

Great news for babies born in with . Now all will be screened at birth so if they have this genetic disease, the family knows so monitoring and interventions can take place to save their life. Great work Eve Salzman Lapin Bobby Lapin!

‘It took a village to achieve’: Texas’ newborn screening lab now testing for ALD The rare genetic disorder occurs primarily in males and affects the nervous system, as well as the adrenal glands.

Wonderful video featuring Maria Kefalas, the founder of The Calliope Joy Foundation, and her amazing drive to help kids with

How One Family Sold Cupcakes to Fund Disease Research Maria Kefalas founded the Calliope Joy Foundation and sold 45,000 cupcakes to start a clinical research center for her daughter's Cal's disease, called leuko...