Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis
(IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions.
is a sort of very heterogeneous disease.
Hapel et al., "Targeted disruption of the mouse colony-stimulating factor 1 receptor gene results in osteopetrosis
, mononuclear phagocyte deficiency, increased primitive progenitor cell frequencies, and reproductive defects," Blood, vol.
The same mutation was also reported with a clinical diagnosis of autosomal recessive osteopetrosis
(ARO) in a patient from Azad Kashmir (Pangrazio et al., 2014).
is a group of genetically heterogeneous diseases that affects bones.
hypophosphatasia, pycnodysostosis and osteopetrosis
) and these conditions should be kept in mind in the differential diagnosis.
Marks Jr., "Osteopetrosis
in the toothless rat: failure of osteoclast differentiation and function," Bone and Mineral, vol.
Autologous bone marrow transplantation has also been used in other pediatric skeletal diseases, such as osteopetrosis
and infantile hypophosphatasia, with promising results [92, 93].
The c-Fos-deficient mice had osteopetrosis
due to a cell-autonomous defect in osteoclast differentiation .
The known clinical conditions such as trauma, bone surgery, or other diseases that can affect the vascularity of bone (osteoporosis, osteopetrosis
, Paget's disease, radiation, and malignancy) and predispose to TBO [28, 29] should be excluded anamnestically.
acro-osteolytica: a syndrome of osteopetrosis
, acro-osteolysis and open sutures of the skull," Acta Chirurgica Scandinavica, vol.