Research Study: Communication and testing experiences of adolescent girls and adult women in families with X-linked adrenoleukodystrophy Purpose of Research: Genetic counselors are often involved in facilitating genetic testing for…
bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD) SKYSONA is the first FDA approved therapy shown to slow the progression of…
Minoryx‘s Marketing Authorization Application for its lead candidate leriglitazone validated by EMA for orphan indication X-linked Adrenoleukodystrophy (X-ALD) 2022-09-14 Mataró, Barcelona, Spain, September 14, 2022 – Minoryx Therapeutics, a late-stage biotech company…
FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy If approved, eli-cel will be the first and only gene therapy for the treatment of early active CALD, a…
SwanBio Therapeutics Announces $56 Million Series B Financing to Advance Novel Gene Therapies for Neurological Conditions New funding will support ongoing clinical development of lead candidate, SBT101 for adrenomyeloneuropathy (AMN)…
Poxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the U.S. FDA for X-Linked Adrenoleukodystrophy LYON, France–(BUSINESS WIRE)– POXEL SA (Euronext: POXEL – FR0012432516), a clinical stage biopharmaceutical company developing innovative…
Natalie Grant presents at 2022 AAN Meeting Natalie Grant, Clinical Research Coordinator, at Massachusetts General Hospital presented, “Neurological Disease Burden in Females with X-Linked Adrenoleukodystrophy” at the 2022 American Academy…
Dr. Jonanlis Ramirez Alcantara presents at the 2022 Pediatric Endocrine Society Meeting Dr. Jonanlis Ramirez Alcantara, Clinical and Research Fellow at Massachusetts General Hospital, presented, “ADRENAL INSUFFICIENCY IN PEDIATRIC PATIENTS…
Cellular, Tissue and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments Original post by the Food and Drug Administration on Apr 13, 2022 The Food…
SwanBio Therapeutics Announces FDA Investigational New Drug Clearance for First AAV-Based Gene Therapy for the Treatment of Adrenomyeloneuropathy Initiation of the Phase 1/2 Trial of SBT101 in Patients with AMN…
bluebird bio Announces FDA Priority Review of Biologics License Application for eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) in Patients Without a Matched Sibling Donor If approved, eli-cel will be…
Poxel Announces the Formation of its Scientific Advisory Board for Rare Metabolic Diseases LYON, France–(BUSINESS WIRE)– POXEL SA (Euronext – POXEL – FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for…
ALD & AMN Cross-Sectional Survey: You can help! Please note: This study is not being conducted by ALD Connect. It is being conducted by Dr. Chad Heatwole from the University…
Poxel Announces its Participation at Upcoming Scientific Conferences Related to Adrenoleukodystrophy (ALD) LYON, France–(BUSINESS WIRE)– POXEL SA (Euronext – POXEL – FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic…
SwanBio Therapeutics Announces New Preclinical Data Demonstrating Proof of Principle for SBT101 Gene Therapy as a Potential Treatment of Adrenomyeloneuropathy SBT101 Demonstrated Dose-Dependent Improvement of AMN Disease Markers in Mouse…
Introducing our new Peer Mentors We are excited to introduce two new Peer Mentors: Tim Mathwig and Brad Gillepsie My name is Tim Mathwig. I am 45 years old and…
Statement from the ALD Connect Board of Directors The ALD Connect Board of Directors released the following statement on August 10, 2021: We would like to acknowledge the recent press…
bluebird bio Reports Second Quarter Financial Results and Provides Operational Update – On track to complete planned business separation in 4Q 2021; each company launching with approximately 24 months of runway…
Autobahn Therapeutics Announces Orphan Drug Designation Granted to ABX-002 for the Treatment of X-linked Adrenoleukodystrophy First-of-its-kind Brain Penetrant Therapy on Track to Enter Clinical Studies in Q4 2021 SAN DIEGO –…
Poxel Announces New Strategic Direction with Increasing Focus on Rare Metabolic Diseases Following Recent Achievements Following the recent approval of TWYMEEG® (Imeglimin) in Japan and associated potential future revenues, Poxel to…
Quality of life in women with X-ALD A study conducted by Dr. Wolfgang Koehler and Dr. Lisa Schäfer on Women with ALD is open and enrolling. Two other studies on Women…
Plasma NfL and GFAP as biomarkers of spinal cord degeneration in adrenoleukodystrophy Plasma NfL and GFAP as biomarkers of spinal cord degeneration in adrenoleukodystrophy
Statement for ALD Patients Considering the COVID-19 Vaccine The ALD Connect Board of Directors released the following statement on December 19, 2020: COVID-19 is a harmful, infectious disease that is…
(This summary of our 2020 ALD Connect Annual Meeting and Patient Learning Academy session on Education Supports for ALD Boys: IEPs AND 504 Plans by Nicole Metsistso Mazer, Esq. was…
Autobahn Therapeutics Completes Successful Pre-IND Meeting with FDA SAN DIEGO–(BUSINESS WIRE)– Autobahn Therapeutics, a biotechnology company focused on restoring hope for people affected by CNS disorders, today announced it has…
Utah newborns now screened for X-linked adrenoleukodystrophy (Salt Lake City, UT) – The Utah Department of Health’s Newborn Screening (NBS) program is excited to announce an additional test for all…
Saskatoon family looking for provincial funding to cover son’s U.S. medical treatment for disease The pandemic has been even more stressful for the Finn family. In mid-June, four-year-old Conner was…
bluebird bio Presents New Results from Clinical Development Program of elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD), Including Updated Long-Term Data, at the 46th Annual Meeting of…
Running miles for ALD: Grafton father’s mission to fund research
ALD Family Weekend – Camp At Home Click here to learn more about ALD Family Weekend.
SwanBio Therapeutics Announces Series A Expansion to Fund its Lead Program and Grow Pipeline in Gene Therapy for Neurological Diseases $52 million is being allocated to progress IND-enabling studies in…
April 2020 Community Calls Symptomatic Women: April 27th at 7:00 PM EST facilitated by Emma Hayes Register here AMN Men: April 28th at 5:00 PM EST facilitated by Tim Maguire and…
Family of boy who became sheriff for a day raising awareness of neurological condition As an honorary Lehigh County Sheriff 8-year-old Kaleb Holder’s energy was highlighted back in March 2016.…
August Community Call “Newborn Screening, Young Families, and Parents of Asymptomatic Children”– Thursday, August 15 2019 @7 PM EST -Facilitated by Suzanne Flynn, Tara Meady, and Kelly Strenges Click here…
ALD Connect Joins Forces with The Myelin Project! We are so excited to announce that ALD Connect will be joining forces with fellow ALD patient organization, The Myelin Project! Check…
July Community Calls Please join us for our July Community Calls! “Symptomatic Women”– Wednesday, July 10th 2019 @7 PM EST -Facilitated by JoAnn Locy and Janis Sherwood Click here to…
New Combination of High-Dose Antioxidants as Therapy for AMN A new trial using high dose antioxidants has improved symptoms in patients with AMN by targeting oxidative stress. Read the abstract…
Emory University Newborn Screening Study Emory University in Georgia is recruiting ALD patients of any age and gender to give a single blood sample to provide quality control for newborn screening…
2019 Annual Meeting Registration Register now for our 2019 Annual Meeting & Patient Learning Academy. Registration will close on October 1st, 2019. Limited financial assistance is available for travel and other costs.…
2018 Annual Meeting ALD Connect 2018 Annual Meeting & Patient Learning Academy Videos 2018 Patient Learning Academy Videos 2018 Annual Meeting Videos Day 1: ALD Connect Consortium Annual Meeting…
Tanay and his family find hope for ALD with Gene Therapy trial Tanay Suri and his family traveled halfway around the world for a groundbreaking adrenoleukodystrophy (ALD) treatment at University…
ALD Comprehensive Clinic Expands to Help More Adrenoleukodystrophy Patients and Families Our center at University of Minnesota Masonic Children’s Hospital has decades of experience treating adrenoleukodystrophy. Now we’re expanding our…
ALD was added to the Recommended Uniform Screening Panel (RUSP), a federal list of all genetic diseases recommended for state newborn screening (NBS) programs, in February 2016. However, at this…
ALD Connect SCOUT program With the creation of the NBS SCOUT Initiative (Supportive Community Outreach & Understanding Together), ALD Connect will draw upon the experience of previously-diagnosed ALD families and…
Parents’ vigilance gives son a fighting chance against Adrenoleukodystrophy 12-year-old Nicholas Purschke plans to run a triathlon this year, a feat that wouldn’t be possible if early screening hadn’t found…
