Results 41 to 50 of about 32,298 (248)

Investigating the landscape of US orphan product approvals

Orphanet Journal of Rare Diseases, 2018
Background The Orphan Drug Act was enacted in 1983 to encourage the development of drugs for rare diseases. Previous research has attempted to examine the impact of the Act by assessing either the number of orphan designations that have been granted or ...
Kathleen L. Miller, Michael Lanthier
doaj   +1 more source

Orphan drugs in Italy: availability and time-to-access at regional level

Global & Regional Health Technology Assessment, 2023
Introduction: There are an estimated 26 million rare disease patients in Europe, about 2 million in Italy. Access to orphan drugs has been evaluated nationally and internationally, and delays have been observed due to evaluation of price and therapeutic ...
Maria Lucia Marino, Eva Alessi, Aurora Di Filippo, Barbara Polistena, Francesco Macchia, Federico Spandonaro, Francesco Trotta   +6 more
doaj   +1 more source

Descriptive Epidemiology From the Myhre Syndrome Foundation Registry: The Value of Self‐Reported Data

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, Armelle Pindon, Maggie R. Brand, Kate Wears, Katherine H. Young, Alyssa Mendel, Michael J. Lyons   +6 more
wiley   +1 more source

Caregiver Reports on the Needs and Experiences of Children Impacted by Parental Incarceration: Results From an Australian Survey

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, Lana Battaglia, Lorana Bartels, Susan Dennison, Susy Harrigan, Lee Huggins, Sofia Grage‐Moore   +6 more
wiley   +1 more source

Patient access to orphan drugs in France

Orphanet Journal of Rare Diseases, 2019
Background Since incentives were introduced to promote orphan drugs in Europe, several dozens of drugs have been registered at the European level. However, patient access on a national level remains very heterogeneous across Europe. This can be explained
Marion Bourdoncle, Blandine Juillard-Condat, Florence Taboulet   +2 more
doaj   +1 more source

ORPHAN DRUG POLICY

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

Efficacy and Safety of Nanoencapsulated Sirolimus Plus Pegadricase: Results From the Randomized, Placebo‐Controlled Phase 3 Trials

Arthritis &Rheumatology, EarlyView.
Objective DISSOLVE I and II examined the efficacy and safety of nanoencapsulated sirolimus (NAS) plus pegadricase (NASP) in patients with uncontrolled gout (UG). Methods In these double‐blind, placebo‐controlled Phase 3 trials of NASP, patients were randomized 1:1:1 to infusions of high‐dose (HD) or low‐dose (LD) NAS plus pegadricase (HD NASP, LD NASP,
Herbert S. B. Baraf, Puja P. Khanna, Milan Petronijevic, Mamuka Lortkipanidze, Anand Patel, Kwabena Ayesu, Michael H. Pillinger, Atul Singhal, Joanna Sobierska, Jacquie Christie, Peter Traber, Rehan Azeem, Wesley DeHaan, Hugues Santin‐Janin, Bhavisha Desai, Alan Kivitz   +15 more
wiley   +1 more source

Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics

British Journal of Clinical Pharmacology, EarlyView.
Abstract This review focuses on the development of radiopharmaceutical imaging agents and radioligand therapeutics for paediatric use. Nuclear medicine plays an important role in the diagnosis and treatment of various childhood conditions, including cancers, infections and brain disorders.
Justin L. Hay, Amandine Manon, Hunter Stephens, S. Y. Amy Cheung, Bart de Keizer, Erik T. te Beek   +5 more
wiley   +1 more source

International experience in drug assessment for rare diseases: flexibility of approaches

Реальная клиническая практика: данные и доказательства
Relevance. The regulation of orphan drugs plays a key role in the development of medicines for rare diseases that affect only a small percentage of the population. This specialized regulatory framework is designed to encourage pharmaceutical companies to
K. S. Radaeva, A. S. Kolbin
doaj   +1 more source

Estimating the budget impact of orphan medicines in Europe: 2010 - 2020

Orphanet Journal of Rare Diseases, 2011
Background Orphan drugs are a growing issue of importance to European healthcare policy makers. The success of orphan drug legislation in Europe has resulted in an increasing number of licensed medicines for rare diseases, and many more yet unlicensed ...
Milanova Tsveta, Schey Carina, Hutchings Adam   +2 more
doaj   +1 more source