Results 121 to 130 of about 88,273 (287)
Children in need of medical innovation [PDF]
Children are therapeutic orphans and an underprivileged group in innovations derived from drug therapy. As the innovation process of pharmaceuticals is a long, risky and very costly business, economists typically emphasise lack of profit incentives and ...
Entorf, Horst +2 more
core
Epilepsia, EarlyView.Soticlestat as adjunctive therapy for Lennox–Gastaut syndrome. Abstract Objective There remains a need for new treatments for Lennox–Gastaut syndrome (LGS), a developmental and epileptic encephalopathy with a heterogenous patient population that often requires polytherapy. The phase 3, randomized SKYWAY study (NCT04938427) investigated the efficacy and
Renzo Guerrini +15 more
wiley +1 more source
Copyright Term Reversion and the “Use-It-Or-Lose-It” Principle [PDF]
, 2012 This brief article makes an argument for the use of the legal device of term reversion, as a means for bringing unexploited works back into use, and mitigating the undesirable effects of the excessive term of copyright protection.
Kretschmer, Martin
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Precision therapies for genetic epilepsies in 2025: Promises and pitfalls
Epilepsia Open, EarlyView.Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang +3 more
wiley +1 more source
When Rare Is Not Small: Amyotrophic Lateral Sclerosis Initiatives and Therapy
Exploration, EarlyView.In the precision‐medicine era, rare diseases must not be sidelined in translational infrastructure. The Mr. Cai Lei—led “Ice‐Breaking Team” turns an amyotrophic lateral sclerosis patient community into a sustainable ecosystem, realigning philanthropy, data, and research and development to reshape rare‐disease pipelines and guide precision therapies ...
Yang Liu +6 more
wiley +1 more source
Transformers: African American Women Leaders in the Pharmaceutical Industry [PDF]
, 2016 This qualitative study documents the experiences of African American women leaders in the pharmaceutical industry in the context of the transformative leadership model. Transformative leadership is a theory that recognizes that the success of individuals
Bonaparte, Yvette Lynne
core +1 more source
Clinical development and marketing application review times for novel orphan-designated drugs
Frontiers in MedicineDevelopment of an orphan-designated drug has been more challenging and financially less attractive than that of other drugs due to low prevalence of the condition, poorly defined biomarkers and lack of experience of healthcare providers in diagnosing and
Ebru Demirci +2 more
doaj +1 more source
The Laryngoscope, EarlyView.ABSTRACT Objective With regulatory approval of HPV‐specific immunotherapy for recurrent respiratory papillomatosis (RRP) and growing experience with systemic bevacizumab, a management algorithm incorporating these medical treatments is warranted. Data Sources and Methods RRP Foundation (RRPF) Key Opinion Leaders offer a proposed management algorithm ...
Simon R. Best +17 more
wiley +1 more source
Movement Disorders Clinical Practice, EarlyView.Abstract Background As Parkinson's disease (PD) progresses, motor fluctuations become increasingly difficult to manage with oral medications. Foslevodopa/foscarbidopa (LDp/CDp), delivered as a continuous 24‐h/day subcutaneous infusion, offers continuous levodopa delivery and stable plasma levodopa levels that reduce motor fluctuations. LDp/CDp has been
K. Ray Chaudhuri +20 more
wiley +1 more source
Analysis of marketed orphan drugs in China. [PDF]
Intractable Rare Dis Res, 2023 Zhi W +5 more
europepmc +1 more source