Orphan Drug Designation - Neonatal Disorders

Relevant Publications

Food and Drug Administration (FDA) Approvals of Biological Drugs in 2023.

Issue Release: 2024

New Drug Development Strategy in Japan: Flexibility in Guideline Adherence.

Issue Release: 2024

Synthetic routes and clinical application of new drugs approved by EMA during 2023.

Issue Release: 2024

A Drug Discovery Perspective on FDA Expedited Development and Incentive Programs.

Issue Release: 2024

The health benefits, costs, and cost-effectiveness of ultra-orphan drugs.

Issue Release: 2024

Toll-like receptors ligand immunomodulators for the treatment congenital diaphragmatic hernia.

Issue Release: 2024

Review of real-world experience with lurbinectedin in relapsed/refractory small cell lung cancer.

Issue Release: 2024

An update patent review of MDM2-p53 interaction inhibitors (2019-2023).

Issue Release: 2024

Real-World Evidence to Support the Registration of a New Osteoporosis Medicinal Product in Europe.

Issue Release: 2024

Diversity Plans and Postmarketing Studies: First Impressions of Anticipated Diversity Requirements in the United States.

Issue Release: 2024

International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea.

Issue Release: 2024

2023 FDA TIDES (Peptides and Oligonucleotides) Harvest.

Issue Release: 2024

Cost of Exempting Sole Orphan Drugs From Medicare Negotiation.

Issue Release: 2024

Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea.

Issue Release: 2024

Factors influencing the reimbursement of cancer drugs in Europe: A scoping review.

Issue Release: 2024

A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases.

Issue Release: 2024

Development and validation of an LC‒MS/MS method for the determination of cyclocreatine phosphate and its related endogenous biomolecules in rat heart tissues.

Issue Release: 2024

Expediting treatments in the 21st century: orphan drugs and accelerated approvals.

Issue Release: 2024

Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease.

Issue Release: 2024

The Prevalence of Diffuse Large B-Cell Lymphoma Varies Substantially by Methods Applied: Results From a Population-Based Study.

Issue Release: 2024

What Types of Martial Arts Are Suitable for Children?

How Can Parents Prepare for Pediatric Appointments?

Why is Understanding Variability Important?

How Does Hydrocortisone Work?

Why is Stem Cell Transplant Important in Neonatal Disorders?

What is Nipple Pain?

How is Dehiscence Diagnosed in Pediatric Patients?

What are the Components?

What is Renal Agenesis?

What Role Does Family Play in Pediatric Care?

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