Tag: "orphan" at medical news
New biotech company to commercialize novel UD gene-repair technology
... in the field. There are more than 5,000 rare or orphan
diseases, so named because each affects fewer than 200,000 people nationwide. A number of these diseases are caused by a single-point mutation in a gene--which is like a spelling error, a single letter out of place, in its DNA code. The DNA nucleotid...
UCSF brain tumor vaccine trial shows promising results
... therapy. Vitespen has been granted fast-track and orphan
drug designations from the Food and Drug Administration for both metastatic melanoma (skin cancer) and renal cell carcinoma (kidney cancer). Reviewing the presentation at AANS, Henry Brem, MD, director of neurosurgery at the Johns Hopkins Medical ...
Vaccine for brain tumors shows promising results
... therapy. Vitespen has been granted fast track and orphan
drug designations from the Food and Drug Administration in both metastatic melanoma (skin cancer) and renal cell carcinoma (kidney cancer). The UCSF clinical trial is a phase1/2 study designed to establish the feasibility, safety and preliminary eff...
Challenges to improving adolescent nutrition in Bangladesh and Tanzania
... shortages, skipping meals, school attendance, and orphan
status," says Levinson. "This data will guide future efforts in Tanzania and permit the development of Tanzanian counseling materials comparable to those used by the programs in Bangladesh." "Increased attention to adolescent malnutrition in dev...
5,000 rare diseases need drugs, but Europe only approves a handful each year
...ar that less stringent criteria are acceptable for orphan
drugs than for drugs for more common diseases, par...t affect less than five people in 10,000. Only 18 orphan
drugs were approved on the basis of epidemiological data, medical plausibility and potential benefit...
Drug aimed at Huntington's eases chorea, the disease's hallmark feature
...e United States, tetrabenazine is designated as an orphan
drug by the FDA since its targeted to a disease directly affecting fewer than 200,000 people in the nation. The symptom that tetrabenazine treats involuntary, writhing movements of the limbs, face, and sometimes the entire body is the hallmark symp...
Free HIV tests cheaper than charging when goal is preventing AIDS
...sed case, counseling and information about HIV and orphan
care and assistance. "They are a remarkable group of women doing the hard work of taking care of AIDS patients for the long-term. They asked us to provide an HIV testing program as a means of promoting behavioral changes, and that's really the genes...
Institute for OneWorld Health receives multimillion dollar grant
...EMEA). In 2005, the FDA and the EMEA each granted orphan
drug status to OneWorld Health for paromomycin for the treatment of VL. Local drug manufacture to GMP standards: OneWorld Health will ensure that paromomycin is manufactured in India using FDA Good Manufacturing Practices, the highest international ...
Clinical trial supports better treatment for lupus nephritis
...n Cytoxan," said Dooley, who helped design the FDA orphan
disease branch-funded study, get it approved and recruit medical centers and patients. "Our results are very promising, but we need to do longer follow-up to see if the new medicine produces long-lasting improvement." Oral mycophenolate mofetil worke...
Synaptic connections need nurturing to retain their structure and keep outsiders at bay
...tic spine (dendrites) of Purkinje cells called the orphan
delta-2 glutamate receptor. They note that models that lack delta-2 glutamate receptor in Purkinje cells are nearly identical to models that lack Cbln1 in granule cells. This suggests that delta-2 glutamate receptor somehow mediates the action of sec...
Should drugs for rare diseases be given special status in the NHS?
...lth services was negligible. But, as more and more orphan
drugs come on to the market, the impact on other h...icle, experts argue that the cost effectiveness of orphan
drugs should be treated in the same way as for other healthcare technologies. They believe that the ...
'Orphan medication' in Europe
...of Medicinal Products (EMEA) has designated, as an orphan
medicine, one of the 18 patents of the Centro de I...A has conceded the simultaneous designation as an orphan
medicine of a product involving two distinct symptoms, thereby endorsing the consistency of the dat...