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Top News In Leukodystrophies Today - Breaking & Trending Today

Two-year-old miracle baby joyfully battles rare brain disease, defying odds

A 2-year-old is doing everything doctors said he would never do again: laugh, see, hear, and even move. ....

United States , Michael Hutchinson , Arthur Hutchinson , Allissa Hutchinson , Krabbe Disease , Globoid Cell , Health Medical Pharma , Saud Allissa , Marc Brown , Rare Diseases ,

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability. ....

Drug Administration , Contract Development , Gene Therapy , Major Histocompatibility Complex , B Cell Maturation Antigen , Inverted Terminal Repeats , Manufacturing Organizations , Good Manufacturing Practice , Gene Therapy , Bone Marrow , Hemophilia B , N Vivo , Sickle Cell Disease , T Cell ,

Remembering Hans: A family's journey raises awareness for rare diseases on Global Peroxisome Biogenesis Disorders Day

October 5th is Global Awareness Day for Peroxisome Biogenesis Disorders, a group of rare and terminal diseases. We one family’s story and how they now advocate ....

South Korea , United States , Hans Ostertag , Jaya Wadhawan , Chris Ostertag , Rachel Ostertag , Global Awareness Day , Peroxisome Biogenesis Disorders , Peroxisome Biogenesis Disorder , Zellweger Spectrum , Health Medical Pharma , Rare Disease , Rare Diseases , Peroxisomal Disorders , Infantile Refsum Disease ,

FDA Fast Tracks Approval of Therapy for Rare Neurologic Disorder

The US Food and Drug Administration has granted accelerated approval to the gene therapy elivaldogene autotemcel (Skysona) for the treatment of cerebral adrenoleukodystrophy in boys. ....

United States , Davida Williams , Elisa Seeger , European Society For Blood , Drug Administration , Medscape Medical , European Society , Bone Marrow , Boston Children , Genomic Medicine , Edicine Genomic , Us Food And Drug Administration , Nited States Food And Drug Administration , Gene Therapy , Precision Medicine , Drug Development ,

FDA Committee Unanimous on Two Gene Therapies

An FDA advisory committee gave a unanimous thumbs up to two new gene therapies, one for beta-thalassemia and the other for a rare but fatal neurologic disorder. ....

Andrew Obenshain , Anne Virginie Eggimann , Drug Administration , Gene Therapies , Genomic Medicine , Edicine Genomic , Biologic Therapy , Gene Therapy , Precision Medicine , Beta Thalassemia , Us Food And Drug Administration , Nited States Food And Drug Administration , Blood Dyscrasia , Hematologic Disorders ,