Results 51 to 60 of about 1,419,402 (298)
American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.ABSTRACT Myhre syndrome is an ultrarare genetic disease characterized by short stature, distinct craniofacial features, cardiovascular and respiratory fibrosis and stenosis, neurodevelopmental delays, autism, intellectual disability, and hearing loss. The natural history of Myhre syndrome is still not fully understood due to a small patient population ...
Mary K. Young +6 more
wiley +1 more source
Weinberg, Joe (Box 91, Folder 22) [PDF]
, 1977 Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core
Pricing and reimbursement of orphan drugs: the need for more transparency
Orphanet Journal of Rare Diseases, 2011 Pricing and reimbursement of orphan drugs are an issue of high priority for policy makers, legislators, health care professionals, industry leaders, academics and patients.
Simoens Steven
doaj +1 more source
PLoS ONE, 2021 BackgroundGlioblastoma (GBM) is the most common malignant brain tumour among adult patients and represents an almost universally fatal disease. Novel therapies for GBM are being developed under the orphan drug legislation and the knowledge on the ...
Pascal Johann, Dominic Lenz, Markus Ries
doaj +1 more source
Innovare Journal of Life Sciences, 2020 Pharmaceutical industry is crucial for mankind and contributes in some way in socio economic development of the society through jobs, community welfare, and supply chains. Indian pharmaceutical companies are one of the largest markets in the world, estimated 10% of global production and 2% of world market.
openaire +3 more sources
Australian Journal of Social Issues, EarlyView.ABSTRACT Children experiencing parental imprisonment are known to be among the most overlooked in our community. They often experience multiple and compounding disadvantages, with long‐term consequences, but receive no specialised assistance. Knowledge about these children and their families is lacking in Australia and is required to inform policy ...
Catherine Flynn +6 more
wiley +1 more source
Lifson, Miriam (Box 91, Folder 5) [PDF]
, 1987 Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core
Bernstein, Edna, Kaminsky, Ceil, and Lit, Sara Liman (Box 90, Folder 8) [PDF]
, 1977 Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core
Incentives for orphan drug research and development in the United States
Orphanet Journal of Rare Diseases, 2008 Background The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions.
Rodriguez-Monguio Rosa +3 more
doaj +1 more source
Badian, Sam (Box 90, Folder 5) [PDF]
, 1976 Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core