Designating an Orphan Product: Drugs and Biological Products

The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor.
Source: FDA Center for Drug Evaluation and Research - What's New - Category: Drugs & Pharmacology Authors: Source Type: news

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Authors: Abstract Here we present the case of a 50-year-old woman with acute urinary retention who was treated by the insertion of a permanent catheter. For associated headaches, fever and muscle and joint pain, the patient underwent neurologic examination, including lumbar puncture and magnetic resonance of head and spine. The results confirmed aseptic meningitis. Subsequently, the patient was hospitalized at the infectious disease clinic, where the permanent catheter was extracted after 5 days, with spontaneous micturition recovery and no post-void residual volume. The combination of aseptic meningitis and urina...
Source: Neuroendocrinology Letters - Category: Endocrinology Tags: Neuro Endocrinol Lett Source Type: research
This study aimed to evaluate fertility in female survivors of childhood DTC by assessing various reproductive characteristics combined with anti-Müllerian hormone (AMH) levels (a marker of ovarian reserve). Methods. Female survivors of childhood DTC, diagnosed at ≤18 years of age between 1970 and 2013 were included. Survivors were excluded when follow-up time was less than 5 years or if they developed other malignancies before or after diagnosis of DTC. Survivors filled out a questionnaire regarding reproductive characteristics (e.g. age at menarche and menopause, pregnancies, pregnancy outcomes, need for assisted ...
Source: Thyroid : official journal of the American Thyroid Association - Category: Endocrinology Tags: Thyroid Source Type: research
In this study, we determined the efficacy of ACTON PROLONGATUM® (AP; Ferring Pharmaceuticals) in comparison with Acthar® Gel (Mallinckrodt) and full 39 amino-acid rat ACTH molecule (Genscript) in the rodent model of IS consisting of prenatal priming with betamethasone and repeated postnatal trigger of spasms with N-methyl-d-aspartate. Treatment with these ACTH varieties was given on postnatal days (P)12, P13, and P14 in a prospective test (treatment onset on P12 AFTER induction of spasms). Two independent arms were investigated: subcutaneous (SC) and intramuscular (IM) deliveries that were evaluated separately. In ...
Source: Epilepsy and Behavior - Category: Neurology Source Type: research
Publication date: Available online 20 February 2020Source: Respiratory InvestigationAuthor(s): Yasuhiro Nakajima, Toshiyuki Minami, Hirotoshi Ishigaki, Eirou Sakai, Ryo Takahashi, Takashi Yokoi, Kozo Kuribayashi, Takashi KijimaAbstractA superior vena cava (SVC) aneurysm is a rare disease that can be confused with upper mediastinal tumor. A 57-year-old female visited our hospital regarding an abnormal shadow in her mediastinum on a chest X-ray. Upon closer examinations, which included three-dimensional computed tomography, we diagnosed it as a SVC aneurysm. Since her SVC aneurysm was regarded as fusiform type at low risk of...
Source: Respiratory Investigation - Category: Respiratory Medicine Source Type: research
FDA thanks the NIH for letting us participate in their 2020 Rare Disease Twitter Chat. #FDARare2020 #NIHchat pic.twitter.com/U3UU87gF7r
Source: Food and Drug Adminstration (FDA): CDRHNew - Category: Medical Devices Authors: Source Type: news
No abstract available
Source: Nursing - Category: Nursing Tags: Department: DRUG CHALLENGE Source Type: research
Are you a rare disease patient, caregiver or advocate with questions or stories to share? Or a researcher with helpful resources? Join us today at 2 pm EST for the @NIH Twitter chat on #RareDiseases. Use #NIHchat to submit your questions. https://go.usa.gov/xpVbT  #RDDNIH pic.twitter.com/WQrYzChIEQ
Source: Food and Drug Adminstration (FDA): CDRHNew - Category: Medical Devices Authors: Source Type: news
FDA is undertaking a number of efforts to advance the development of medical products to meet the needs of the rare disease population.
Source: FDA Center for Drug Evaluation and Research - What's New - Category: Drugs & Pharmacology Authors: Source Type: news
AbstractBackgroundLipodystrophies are a group of diseases which are characterized by abnormal adipose tissue deposition and are frequently associated with metabolic changes. Congenital generalized lipodystrophy is an autosomal recessive syndrome, with a prevalence
Source: Journal of Medical Case Reports - Category: General Medicine Source Type: research
The FDA works with many people and groups, such as patients, caregivers, and drug and device manufactures, to support rare disease product development.
Source: FDA Center for Drug Evaluation and Research - What's New - Category: Drugs & Pharmacology Authors: Source Type: news
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