| P50 | author | Antonello Di Paolo | Q42529822 |
| P2093 | author name string | Elena Arrigoni | |
| P2860 | cites work | Glycobiology | Q22255657 |
| | Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries | Q26785430 |
| | Effectiveness, safety and costs of orphan drugs: an evidence-based review | Q26827237 |
| | Delayed access to treatments for rare diseases: who's to blame? | Q26864797 |
| | Changing the cost of care for chronic myeloid leukemia: the availability of generic imatinib in the USA and the EU | Q28086893 |
| | A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases | Q28650616 |
| | Generic oncology drugs: are they all safe? | Q30241714 |
| | Is extrapolation of the safety and efficacy data in one indication to another appropriate for biosimilars? | Q30675199 |
| | Orphan therapies: making best use of postmarket data | Q30836776 |
| | Clinical data and regulatory issues of biosimilar products | Q31038317 |
| | Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007-13) and prospective (2014-18) MIDAS sales data analysis | Q31098648 |
| | Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry | Q33438029 |
| | Potential Clinical and Economic Impact of Switching Branded Medications to Generics | Q33637817 |
| | Gastrointestinal manifestations of endocrine disease | Q33868377 |
| | Biosimilar epoetins and other "follow-on" biologics: update on the European experiences | Q34131083 |
| | Developmental pharmacology--drug disposition, action, and therapy in infants and children | Q34247047 |
| | Orphan drugs expenditure in the Netherlands in the period 2006-2012. | Q34333608 |
| | Clinical pharmacokinetics of sirolimus | Q34347243 |
| | Cognitive and affective determinants of generic drug acceptance and use: cross-sectional and experimental findings | Q34908974 |
| | Developing a patient-directed policy framework for managing orphan and ultra-orphan drugs throughout their lifecycle | Q35043701 |
| | Cyclosporine: A Commentary on Brand versus Generic Formulation Exchange | Q35607575 |
| | The supportive care needs of parents with a child with a rare disease: results of an online survey. | Q36083344 |
| | Fully Synthetic Granulocyte Colony-Stimulating Factor Enabled by Isonitrile-Mediated Coupling of Large, Side-Chain-Unprotected Peptides | Q36198586 |
| | Drugs for exceptionally rare diseases: do they deserve special status for funding? | Q36276435 |
| | Generic substitution: the use of medicinal products containing different salts and implications for safety and efficacy | Q36368734 |
| | Are there potential problems with generic substitution of antiepileptic drugs? A review of issues | Q36407768 |
| | Generic and brand-name L-thyroxine are not bioequivalent for children with severe congenital hypothyroidism | Q36589423 |
| | Impact of orphan drugs on Latvian budget | Q36895393 |
| | The challenge of biosimilars | Q36943139 |
| | The first biosimilar epoetin: but how similar is it? | Q37023552 |
| | Difficulties in the production of identical drug products from a pharmaceutical technology viewpoint. | Q37093853 |
| | Novel bioequivalence approach for narrow therapeutic index drugs | Q41452460 |
| | O-linked sugar chain of human granulocyte colony-stimulating factor protects it against polymerization and denaturation allowing it to retain its biological activity | Q41728167 |
| | Safety considerations of biosimilars | Q42353230 |
| | Rare diseases, orphan drugs and their regulation: questions and misconceptions | Q43814369 |
| | Why we should care about ultra-rare disease | Q44040612 |
| | Bioequivalence and other unresolved issues in generic drug substitution | Q44704654 |
| | Care for patients with ultra-rare disorders | Q45246075 |
| | Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. | Q46099441 |
| | Microheterogeneity of erythropoietin carbohydrate structure | Q46150335 |
| | What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis | Q46689856 |
| | Altered steady state pharmacokinetics of levofloxacin in adult cystic fibrosis patients receiving calcium carbonate. | Q46946944 |
| | Repurposing of Proton Pump Inhibitors as first identified small molecule inhibitors of endo-β-N-acetylglucosaminidase (ENGase) for the treatment of NGLY1 deficiency, a rare genetic disease | Q47575481 |
| | Strengths, weaknesses and future challenges of biosimilars' development. An opinion on how to improve the knowledge and use of biosimilars in clinical practice | Q47637542 |
| | Molecular and clinical characterization of a series of patients with childhood-onset lysosomal acid lipase deficiency. Retrospective investigations, follow-up and detection of two novel LIPA pathogenic variants | Q48020927 |
| | Orphan drug policies and use in pediatric nephrology | Q48302245 |
| | International Consensus Statement on the Clinical and Therapeutic Management of Leber Hereditary Optic Neuropathy | Q48577544 |
| | Gastrointestinal Symptoms in Children and Adolescents With Neurofibromatosis Type 1. | Q49842740 |
| | Scientific considerations for assessing biosimilar products | Q50429210 |
| | Biosimilar recombinant human erythropoietin induces the production of neutralizing antibodies. | Q50541835 |
| | Therapeutic monitoring of pediatric renal transplant patients with conversion to generic cyclosporin. | Q51083518 |
| | Therapeutic profile of orphan medicines. | Q51787377 |
| | Clinical efficacy of generic imatinib | Q57108016 |
| | Rare diseases and orphan drugs | Q64040266 |
| | The Pediatric Research Equity Act Moves Into Adolescence | Q64047516 |
| | Gaucher disease: recommendations on diagnosis, evaluation, and monitoring | Q77302755 |
| | Withdrawal of generic budeprion for nonbioequivalence | Q85631225 |
| | Lymphangioleiomyomatosis | Q88106408 |
| | Generic antiretrovirals for the treatment of HIV: a novel challenge for Western countries? | Q95480370 |
| | Systematic review of available evidence on 11 high-priced inpatient orphan drugs. | Q37116422 |
| | Why rare diseases are an important medical and social issue | Q37191857 |
| | Anticancer drug therapy in the older cancer patient: pharmacology and polypharmacy | Q37229270 |
| | Drug development for exceptionally rare metabolic diseases: challenging but not impossible | Q37350225 |
| | Factors and Mechanisms for Pharmacokinetic Differences between Pediatric Population and Adults | Q37378093 |
| | Idebenone: an emerging therapy for Friedreich ataxia | Q37414033 |
| | Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs? | Q37566934 |
| | Estimating the budget impact of orphan drugs in Sweden and France 2013-2020. | Q37595752 |
| | Translation of rare disease research into orphan drug development: disease matters | Q37612173 |
| | Gastrointestinal manifestations of mitochondrial disorders: a systematic review | Q37671957 |
| | Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL). | Q37692633 |
| | Clinical concerns of immunogenicity produced at cellular levels by biopharmaceuticals following their parenteral administration into human body | Q37700537 |
| | From promising molecules to orphan drugs: Early clinical drug development | Q37712671 |
| | Biosimilars: controversies as illustrated by rhGH. | Q37714921 |
| | Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years | Q37763492 |
| | The nocebo effect: a reason for patients' non-adherence to generic substitution? | Q37775939 |
| | Pharmacokinetic evaluation of idebenone | Q37800933 |
| | "Creating hope" and other incentives for drug development for children | Q37830230 |
| | Drugs and mitochondrial diseases: 40 queries and answers. | Q37980195 |
| | Orphan drugs: the regulatory environment | Q38043761 |
| | Development and regulation of biosimilars: current status and future challenges. | Q38095808 |
| | Orphan drugs policies: a suitable case for treatment | Q38179281 |
| | New regulatory paradigms for innovative drugs to treat pediatric diseases. | Q38258387 |
| | Toward a functional definition of a "rare disease" for regulatory authorities and funding agencies. | Q38286754 |
| | Generics Substitution, Bioequivalence Standards, and International Oversight: Complex Issues Facing the FDA. | Q38410842 |
| | Generic immunosuppression in solid organ transplantation: systematic review and meta-analysis. | Q38535040 |
| | Estimating the budget impact of orphan medicines in Europe: 2010 - 2020. | Q38680339 |
| | Epidemiology of mucopolysaccharidoses. | Q38735623 |
| | Trends In Orphan New Molecular Entities, 1983-2014: Half Were First In Class, And Rare Cancers Were The Most Frequent Target | Q38765034 |
| | Experiences of caregivers of children with inherited metabolic diseases: a qualitative study | Q38786033 |
| | Idebenone: A Review in Leber's Hereditary Optic Neuropathy | Q38806554 |
| | The effect of generic switching on concerns about medicine and non-persistence among Danish adults in a general practice setting. | Q38956089 |
| | Generic medicines: solutions for a sustainable drug market? | Q39386312 |
| | Switching Stable Kidney Transplant Recipients to a Generic Tacrolimus Is Feasible and Safe, but It Must Be Monitored. | Q40313782 |
| | Transition from brand to generic tacrolimus is associated with a decrease in trough blood concentration in pediatric heart transplant recipients | Q40396722 |
| | Is age a risk factor for liver disease and metabolic alterations in ataxia Telangiectasia patients? | Q41263470 |
| P921 | main subject | rare disease | Q929833 |
| | orphan drug | Q1367466 |
| P577 | publication date | 2018-02-20 | |
| P1433 | published in | Drugs | Q3040094 |
| P1476 | title | Generic Substitution of Orphan Drugs for the Treatment of Rare Diseases: Exploring the Potential Challenges | |