Rocket's first two clinical programmes using LVV-based gene therapy are for the treatment of Fanconi Anemia, a difficult to treat genetic disease that leads to
bone marrow failure and potentially cancer, and Leukocyte Adhesion Deficiency-I (LAD-I), a serve pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal.
Fanconi Anemia (FA) is a rare pediatric disease characterized by
bone marrow failure, malformations and cancer predisposition.
The genetic disorder causes
bone marrow failures and leads to a predisposition for cancers, and can cause birth and developmental defects.
Myeloid Neoplasm Associated With Inherited
Bone Marrow Failure Syndromes and Telomere Biology Disorders
The goal is to translate the knowledge into treatments for both Fanconi anemia and for the broader problem of
bone marrow failure.
She is fighting a rare
bone marrow failure disorder
Gemahsis' devastated mother Tamar Srondy has told how her young boy was diagnosed with a disease called dyskeratosis congenita - rare and progressive
bone marrow failure syndrome.
Dyskeratosis congenita or Zinsser-Cole-Engman syn-dromeis is a rare inherited
bone marrow failure syndrome (IBMFS) characterized by diagnostic triad of reticulated skin hyperpigmentation, nail dystrophy, and oromucosal leukoplakia (1).
Aplastic anemia is a syndrome of
bone marrow failure characterized by peripheral pancytopenia and marrow hypoplasia.
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Bone marrow failure. With old age there is loss of some of the stem cells.
Among those with high-intensity conditioning, malignancy and
bone marrow failure were the most common indications (seven patients each); one patient had an immunodeficiency, and the cause was unknown for another.
Cyclosporine and ATG may improve hepatitis as well as
bone marrow failure via suppression of cytotoxic T lymphocytes [19].
The inherited
bone marrow failure (BMF) syndromes are a group of disorders usually diagnosed in childhood and passed down from parent to child through the association with some genetic abnormality [9], which may cause the aplastic anemia (AA) and cancer predisposition [10].
A public health expert Dr Wasim Khawaja from Pakistan Institute of Medical Sciences (PIMS) said that the complications of dengue fever are depression, chronic fatigue, pneumonia,
bone marrow failure, iritis, orchitis, and variety of neurological complications.
Al-Ibrahimi conducted successful bone marrow transplants on 12 Bahraini children in Jordan over the past years -- 2 cases of leukemia, 5 cases of Thalassemia, 1 case of
bone marrow failure syndrome, 1 case of Bone marrow fibrosis, 1 case of Gideak Hakashy Syndrome and one case of Autologous bone marrow transplant.
