Drug development challenges and strategies to address emerging and resistant fungal pathogens

The cost of treating mucormycosis with isavuconazole compared with standard therapy in the UK

(2017) Emma Bagshaw et al.   Future Microbiology

Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for a patient presenting with a PRKAG2 mutation

(2017) Stephanie L. Austin et al.   MOLECULAR GENETICS AND METABOLISM

The emerging phenotype of late-onset Pompe disease: A systematic literature review

(2017) Justin Chan et al.   MOLECULAR GENETICS AND METABOLISM

Polar Extremes in the Clinical Use of Stem Cells

(2017) George Q. Daley   NEW ENGLAND JOURNAL OF MEDICINE

Invasive pulmonary aspergillosis in an immunocompromised patient with severe ulcerative colitis

(2017) David Marti Aguado et al.   REVISTA ESPANOLA DE ENFERMEDADES DIGESTIVAS

Nosocomial fungemia by Candida auris: First four reported cases in continental Europe

(2017) Alba Cecilia Ruiz Gaitán et al.   REVISTA IBEROAMERICANA DE MICOLOGIA

Time to Review the Role of Surrogate End Points in Health Policy: State of the Art and the Way Forward

(2017) Oriana Ciani et al.   VALUE IN HEALTH

Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies

(2017) Regina El Dib et al.   PLoS One

Azole-resistant Aspergillus fumigatus harboring TR34/L98H, TR46/Y121F/T289A and TR53 mutations related to flower fields in Colombia

(2017) Carlos Alvarez-Moreno et al.   Scientific Reports

Investigation of the First Seven Reported Cases ofCandida auris,a Globally Emerging Invasive, Multidrug-Resistant Fungus-United States, May 2013-August 2016

(2016) S. Vallabhaneni et al.   AMERICAN JOURNAL OF TRANSPLANTATION

Using Bayesian Adaptive Trial Designs for Comparative Effectiveness Research: A Virtual Trial Execution

(2016) Bryan R. Luce et al.   ANNALS OF INTERNAL MEDICINE

Prevalence, clinical and economic burden of mucormycosis-related hospitalizations in the United States: a retrospective study

(2016) Dimitrios P. Kontoyiannis et al.   BMC INFECTIOUS DISEASES

Evaluation of a multi-arm multi-stage Bayesian design for phase II drug selection trials – an example in hemato-oncology

(2016) Louis Jacob et al.   BMC Medical Research Methodology

Emergence ofCandida auris: An International Call to Arms

(2016) Cornelius J. Clancy et al.   CLINICAL INFECTIOUS DISEASES

Simultaneous Emergence of Multidrug-ResistantCandida aurison 3 Continents Confirmed by Whole-Genome Sequencing and Epidemiological Analyses

(2016) Shawn R. Lockhart et al.   CLINICAL INFECTIOUS DISEASES

Engaging Participants in Rare Disease Research: A Qualitative Study of Duchenne Muscular Dystrophy

(2016) Roxanna M. Bendixen et al.   CLINICAL THERAPEUTICS

Treatment of mucormycosis in transplant patients

(2016) Maria N. Chitasombat et al.   CURRENT OPINION IN INFECTIOUS DISEASES

Olaratumab: First Global Approval

(2016) Matt Shirley   DRUGS

ECIL-6 guidelines for the treatment of invasive candidiasis, aspergillosis and mucormycosis in leukemia and hematopoietic stem cell transplant patients

(2016) Frederic Tissot et al.   HAEMATOLOGICA

Data Standardization, Pharmaceutical Drug Development, and the 3Rs

(2016) Laura Kaufman et al.   ILAR JOURNAL

Monotherapy or combination therapy of isavuconazole and micafungin for treating murine mucormycosis

(2016) Teclegiorgis Gebremariam et al.   JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY

Phase 3 pharmacokinetics and safety study of a posaconazole tablet formulation in patients at risk for invasive fungal disease

(2016) Oliver A. Cornely et al.   JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY

AR-12 Inhibits Multiple Chaperones Concomitant With Stimulating Autophagosome Formation Collectively Preventing Virus Replication

(2016) Laurence Booth et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Can Multidrug-Resistant Candida auris Be Reliably Identified in Clinical Microbiology Laboratories?

(2016) Masako Mizusawa et al.   JOURNAL OF CLINICAL MICROBIOLOGY

Multidrug-resistant Candida auris : ‘new kid on the block’ in hospital-associated infections?

(2016) A. Chowdhary et al.   JOURNAL OF HOSPITAL INFECTION

Randomized trial of micafungin versus fluconazole as prophylaxis against invasive fungal infections in hematopoietic stem cell transplant recipients

(2016) Silvia Park et al.   JOURNAL OF INFECTION

First report of Candida auris in America: Clinical and microbiological aspects of 18 episodes of candidemia

(2016) Belinda Calvo et al.   JOURNAL OF INFECTION

Identification of Uncommon Candida Species Using Commercial Identification Systems

(2016) Tae-Hyoung Kim et al.   JOURNAL OF MICROBIOLOGY AND BIOTECHNOLOGY

Isavuconazole treatment for mucormycosis: a single-arm open-label trial and case-control analysis

(2016) Francisco M Marty et al.   LANCET INFECTIOUS DISEASES

Research methods to change clinical practice for patients with rare cancers

(2016) Lucinda Billingham et al.   LANCET ONCOLOGY

The anti-Aspergillusdrug pipeline: Is the glass half full or empty?: Table 1.

(2016) Nir Osherov et al.   MEDICAL MYCOLOGY

Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years

(2016) Karolina M. Stepien et al.   MOLECULAR GENETICS AND METABOLISM

Data Sharing

(2016) Dan L. Longo et al.   NEW ENGLAND JOURNAL OF MEDICINE

From Patient to Patient — Sharing the Data from Clinical Trials

(2016) Charlotte J. Haug   NEW ENGLAND JOURNAL OF MEDICINE

Data Sharing and Inductive Learning — Toward Healthy Birth, Growth, and Development

(2016) N. L’ntshotsholé Jumbe et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Global, Neutral Platform for Sharing Trial Data

(2016) Barbara E. Bierer et al.   NEW ENGLAND JOURNAL OF MEDICINE

Avoiding Data Dumpsters — Toward Equitable and Useful Data Sharing

(2016) Laura Merson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Agua potable como posible fuente de brote de diarrea por Microsporidium spp. en pacientes inmunocomprometidos en un hospital pediátrico

(2016) Paulina Coria et al.   Revista Chilena de Infectologia

ECIL-6 guidelines for the treatment of invasive candidiasis, aspergillosis and mucormycosis in leukemia and hematopoietic stem cell transplant patients

(2016) Frederic Tissot et al.   HAEMATOLOGICA

Role of isavuconazole in the treatment of invasive fungal infections

(2016) Dustin Wilson et al.   Therapeutics and Clinical Risk Management

Combination Antifungal Therapy for Invasive Aspergillosis

(2015) Kieren A. Marr et al.   ANNALS OF INTERNAL MEDICINE

Antifungal Activities of SCY-078 (MK-3118) and Standard Antifungal Agents against Clinical Non-Aspergillus Mold Isolates

(2015) Frédéric Lamoth et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Clinical trial designs for rare diseases: Studies developed and discussed by the International Rare Cancers Initiative

(2015) Jan Bogaerts et al.   EUROPEAN JOURNAL OF CANCER

New drug on the horizon for treating adenovirus

(2015) William SM Wold et al.   EXPERT OPINION ON PHARMACOTHERAPY

Más allá de compartir los datos

(2015) Rosa Lamarca et al.   MEDICINA CLINICA

Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints

(2015) Emil D Kakkis et al.   Orphanet Journal of Rare Diseases

How to bolster the antifungal pipeline

(2015) D. W. Denning et al.   SCIENCE

Assessing site performance in the Altair study, a multinational clinical trial

(2015) Nisha Berthon-Jones et al.   Trials

Trends in utilization of FDA expedited drug development and approval programs, 1987-2014: cohort study

(2015) Aaron S Kesselheim et al.   BMJ-British Medical Journal

Precompetitive Data Sharing as a Catalyst to Address Unmet Needs in Parkinson’s Disease 1

(2015) Diane Stephenson et al.   Journal of Parkinsons Disease

Data quality monitoring and performance metrics of a prospective, population-based observational study of maternal and newborn health in low resource settings

(2015) Shivaprasad S Goudar et al.   Reproductive Health

Trends in utilization of FDA expedited drug development and approval programs, 1987-2014: cohort study

(2015) Aaron S Kesselheim et al.   BMJ-British Medical Journal

(1→3)-β-d-Glucan in Cerebrospinal Fluid as a Biomarker forCandidaandAspergillusInfections of the Central Nervous System in Pediatric Patients

(2015) Christine M. Salvatore et al.   Journal of the Pediatric Infectious Diseases Society

How Drugs are Developed and Approved by the FDA: Current Process and Future Directions

(2014) Arthur A Ciociola et al.   AMERICAN JOURNAL OF GASTROENTEROLOGY

Pharmacodynamic Target Evaluation of a Novel Oral Glucan Synthase Inhibitor, SCY-078 (MK-3118), Using anIn VivoMurine Invasive Candidiasis Model

(2014) Alexander J. Lepak et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Methylprednisolone Enhances the Growth of Exserohilum rostratum In Vitro, Attenuates Spontaneous Apoptosis, and Increases Mortality Rates in Immunocompetent Drosophila Flies

(2014) Dimitrios Farmakiotis et al.   JOURNAL OF INFECTIOUS DISEASES

Exserohilum infections: Review of 48 cases before the 2012 United States outbreak

(2014) A. Katragkou et al.   MEDICAL MYCOLOGY

Emerging drugs and vaccines for Candidemia

(2014) Brad Moriyama et al.   MYCOSES

Mold Infections of the Central Nervous System

(2014) Matthew McCarthy et al.   NEW ENGLAND JOURNAL OF MEDICINE

Strategic Biomarkers for Drug Development in Treating Rare Diseases and Diseases in Neonates and Infants

(2013) Jane P. F. Bai et al.   AAPS Journal

Developing Standards for Breakthrough Therapy Designation in Oncology

(2013) S. J. Horning et al.   CLINICAL CANCER RESEARCH

What Can We Learn and What Do We Need to Know Amidst the Iatrogenic Outbreak of Exserohilum Rostratum Meningitis?

(2013) D. P. Kontoyiannis et al.   CLINICAL INFECTIOUS DISEASES

Gene therapy on the move

(2013) Kerstin B. Kaufmann et al.   EMBO Molecular Medicine

Clinical Trials in Rare Disease

(2013) Erika F. Augustine et al.   JOURNAL OF CHILD NEUROLOGY

Practical Aspects of Recruitment and Retention in Clinical Trials of Rare Genetic Diseases: The Phenylketonuria (PKU) Experience

(2013) Stephanie J. DeWard et al.   Journal of Genetic Counseling

Providing guidance in the dark: rare renal diseases and the challenge to improve the quality of evidence

(2013) D. Bolignano et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Review of Clinical Trials for Mitochondrial Disorders: 1997–2012

(2013) Douglas S. Kerr   Neurotherapeutics

Drug development for exceptionally rare metabolic diseases: challenging but not impossible

(2013) Michelle Putzeist et al.   Orphanet Journal of Rare Diseases

A Pilot Study of Multicriteria Decision Analysis for Valuing Orphan Medicines

(2013) Jon Sussex et al.   VALUE IN HEALTH

Epidemiology and Clinical Manifestations of Mucormycosis

(2012) George Petrikkos et al.   CLINICAL INFECTIOUS DISEASES

Fungal Infections Associated with Contaminated Methylprednisolone in Tennessee

(2012) Marion A. Kainer et al.   NEW ENGLAND JOURNAL OF MEDICINE

The rare disease challenge and how to promote a productive rare disease community: Case study of Birt-Hogg-Dubé Symposia

(2012) Vicki L Colledge et al.   Orphanet Journal of Rare Diseases

Adaptive trial designs: a review of barriers and opportunities

(2012) John A Kairalla et al.   Trials

Frequentist and Bayesian Pharmacometric-Based Approaches To Facilitate Critically Needed New Antibiotic Development: Overcoming Lies, Damn Lies, and Statistics

(2011) Paul G. Ambrose et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Additional safety risk to exceptionally approved drugs in Europe?

(2011) Arna H. Arnardottir et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Adaptive design clinical trials: Methodology, challenges and prospect

(2010) Rajiv Mahajan et al.   INDIAN JOURNAL OF PHARMACOLOGY

The role and interpretation of pilot studies in clinical research

(2010) Andrew C. Leon et al.   JOURNAL OF PSYCHIATRIC RESEARCH

Galactomannan serves as a surrogate endpoint for outcome of pulmonary invasive aspergillosis in neutropenic hematology patients

(2009) Johan Maertens et al.   CANCER

Phaeohyphomycosis in a Tertiary Care Cancer Center

(2009) Ronen Ben‐Ami et al.   CLINICAL INFECTIOUS DISEASES

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Predictors for clinical trial participation in the rare lung disease lymphangioleiomyomatosis

(2009) Brent W. Kinder et al.   RESPIRATORY MEDICINE

Clinical research for rare disease: Opportunities, challenges, and solutions

(2008) Robert C. Griggs et al.   MOLECULAR GENETICS AND METABOLISM

Early diagnosis of fungal infection in immunocompromised patients

(2007) R. A. Barnes   JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY