Results 131 to 140 of about 88,273 (287)
Does Misery Love Company? Evidence from pharmaceutical markets before and after the Orphan Drug Act [PDF]
With substantial fixed costs of drug development, more common conditions can support more products. If additional pharmaceutical products are beneficial, they will attract greater consumption and promote better health, e.g. greater longevity.
Frank R. Lichtenberg, Joel Waldfogel
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Movement Disorders Clinical Practice, EarlyView.Abstract Background Microscopic colitis (MC) typically presents with chronic, non‐bloody watery diarrhea. Diagnosis requires endoscopy with colonic mucosal biopsies. The etiology is multifactorial, with several medications implicated, although only a few cases have been attributed to oral levodopa/dopa‐decarboxylase inhibitor (LDDCI) therapy.
Romana Hintner +10 more
wiley +1 more source
Arbor: Ciencia, Pensamiento y Cultura, 2018 Orphan drugs are intended for the treatment of diseases of low prevalence, also known as rare diseases. Research and development into new therapies for this heterogeneous group of diseases presents a number of well-recognized difficulties. One of these is that the small number of patients affected jeopardizes the economic return on the investment made ...
Fontanet Sacristán, Juan Manuel +1 more
openaire +1 more source
Regulating Innovative Medicine: Fitting Square Pegs in Round Holes [PDF]
, 2005 Increasingly, innovative medical products are creating a quandary for the Food and Drug Administration ( FDA ) because they often transcend the FDA\u27s traditional categorical approach to regulating medical products. In a recent attempt to simplify this
Lavender, Mark
core +1 more source
Med Research, EarlyView.ABSTRACT Circadian rhythms orchestrate cardiovascular physiology by regulating immune and inflammatory pathways. Disruption of these rhythms profoundly alters vascular homeostasis, thereby promoting the onset and progression of cardiovascular diseases (CVD).
Tian Zhang +22 more
wiley +1 more source
International Journal for Equity in HealthObjective The accessibility issue of orphan drugs in China is prominent. Based on real-world data from a tier-one city in Northeast China, this study aims to analyze the current usage and affordability of orphan drugs for rare diseases.
Yaqun Wang +6 more
doaj +1 more source
Assessment of the availability of repurposed orphan drugs in India. [PDF]
PLOS Glob Public Health, 2023 Rajueni K, Chakraborty Choudhury M.
europepmc +1 more source
Muscle &Nerve, EarlyView.This exploratory analysis of data from the NEURO‐TTRansform trial evaluated sex‐specific responses in patients with ATTRv‐PN who had received eplontersen in NEURO‐TTRansform versus a historical placebo. Eplontersen can lead to substantial decreases in percentage serum TTR levels, arrest neuropathy impairment, and improve patient quality of life ...
Márcia Waddington Cruz +11 more
wiley +1 more source
From HBV to MASLD Cirrhosis: Mechanistic Insights and Therapeutic Strategies
Portal Hypertension &Cirrhosis, EarlyView.This review examines the epidemiological shift from hepatitis B virus (HBV) to metabolic dysfunction‐associated steatotic liver disease (MASLD) as the leading cause of cirrhosis globally. It highlights the distinct pathogenic mechanisms between HBV and MASLD cirrhosis and discusses evolving diagnostic tools and therapeutic strategies tailored to the ...
Hanqi Yu +5 more
wiley +1 more source
Two-stage public-private partnership proposal for R&D on neglected diseases [PDF]
We propose a 2-stage procurement model of public-private partnership to provide better incentives for R&D on neglected diseases. The model combines advance market commitment, subsidized clinical trials, and rewards based on therapeutical contributions of
Brigitte Granville, Eshref Trushin
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