VANCOUVER — Autologous hematopoietic stem cell (HSC) gene therapy may offer a safe and effective alternative to allogeneic bone marrow transplant for patients with cerebral adrenoleukodystrophy, ...
This rare and devastating genetic disease can now be effectively treated with haploidentical stem cell transplantation.
NeuroVia Closes Series A Financing Round of $14M to Develop Novel Treatment Option for X-ALD Disease
CAMBRIDGE, Mass.--(BUSINESS WIRE)--NeuroVia, Inc., a biopharmaceutical company focused on developing innovative therapies for rare genetic neurological diseases, announced the closing of a Series A ...
Please provide your email address to receive an email when new articles are posted on . Most patients who received Skysona for cerebral adrenoleukodystrophy did not have major functional disabilities ...
THE GROUND BY SUNDAY AFTERNOON. ALL RIGHT. THANK YOU OLGA. ALDI IS A RARE GENETIC DISEASE THAT AFFECTS 1 IN 17,000 PEOPLE WORLDWIDE. THE DIAGNOSIS HIT ONE FAMILY WHO IS ORIGINALLY FROM ALBUQUERQUE.
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