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…
