CDC Director Thomas Frieden said much work remains in the fight against Zika, including improved diagnostics, vaccine development, mosquito control and studies on Zika's long-term effects on pregnant women, men and infants born to Zika-infected mothers. He said he hopes Congress will approve the proposed $300 million Rapid Reserve Fund that would allow the government to act quickly in the event of an infectious disease emergency.
An experimental therapy tested in mice is now being tested in a few children with Sanfilippo syndrome, a rare and debilitating genetic disorder that affects children. A single dose tested in mice restored enzyme activity, increased life span, and improved cognitive and physiological function.
Oral selumetinib shrank tumors in 17 of 24 children with neurofibromatosis type 1, a genetic condition that causes disfiguring but benign tumors of the peripheral nerves, researchers reported. Previous experiments in mice showed that the drug works by interfering with signaling pathways that regulate tumor growth.
A study of mice at Cincinnati Children's Hospital Medical Center found that over-expression of the TRAF6 protein in blood cells led to bone marrow failure and impaired formation of blood cells, researchers reported in the journal Nature Immunology. The findings could lead to the development of possible treatment targets for myelodysplasia syndromes, according to the study.
An experimental drug developed as a male contraceptive blocks sperm maturation and appears to shrink cancerous testicular tumors. The drug, JQ1, modifies gene expression and prompted apoptosis of tumor cells in mouse models alone and in combination with romidepsin, according to a study in the Journal of Cellular and Molecular Medicine.
One in six dogs has significant itching, and for 15% to 20% of them, atopic dermatitis is the diagnosis. In recent years, veterinarians have started turning to new drugs that offer better results than earlier treatments with fewer side effects.
The standards for clinical trials can be flexible, as in the case of the recently approved Duchenne muscular dystrophy drug, but evidence of effectiveness as well as safety is required by law, said FDA Commissioner Dr. Robert Califf. The FDA's biggest challenges are dealing with the growing complexity of data and the need to make decisions with limited information, he said.
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