Rare and Neglected Diseases Archives

Case Study

利用一种罕见病的临床数据支持另一种罕见病的药物审批

Atypical hemolytic uremic syndrome (aHUS) is an ultra-rare genetic disease that causes abnormal blood clot…

Becca Bucci2022 年 6 月 1 日

Blog

A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease

New therapeutics discovery and development for ocular diseases have been traditionally associated with a low…

Jim Gallagher2021 年 12 月 15 日

Case Study

Dose Optimization Using Population PK for an Orphan Drug

RAPALIMUS® Tablets (sirolimus) is Nobelpharma’s orphan drug developed from an oral medication that is sold…

Jim Gallagher2021 年 11 月 17 日

On-Demand Webinar

罕见病管理的“可怕”未来

In a world of rising healthcare costs, the greatest nightmare for payers is reimbursing drug…

Jim Gallagher2021 年 9 月 23 日

Case Study

Using a PBPK-PD Receptor Occupancy Model to Determine the Optimal Dose of Radiprodil for Neonates

Radiprodil is a selective allosteric modulator of the NR2B N-methyl-D-aspartate receptor GluN2B-NMDA that was being…

Jim Gallagher2021 年 8 月 26 日

Case Study

Simcyp’s Age-Defining Feature Facilitates Everolimus Dosing in Neonates

Everolimus (Afinitor®) was recently approved as adjunctive therapy for tuberous sclerosis complex (TSC)-associated partial seizures…

Jim Gallagher2021 年 8 月 26 日

Case Study

Eliglustat for Gaucher Disease: Quantifying the Impact of Pharmacogenetic Status on DDIs

In 2014, Eliglustat (Cerdelga®) was approved by the FDA as the first long-term treatment for…

Jim Gallagher2021 年 8 月 26 日

Case Study

Using PBPK for Label Recommendations in Rare Disease: Deflazacort for Duchenne Muscular Dystrophy

Jim Gallagher2021 年 8 月 20 日

Case Study

使用 Simcyp 模拟器的 DDI 监管海报：Ibrutinib (Imbruvica®)

Pharmacylics and J&J sought to bring ibrutinib, its new tyrosine kinase inhibitor therapy targeting rare…

Jim Gallagher2021 年 7 月 29 日

Case Study

治疗镰状细胞病的 Voxelotor：无需任何临床研究，利用剂量预测模型进行 DDI 预测

In November 2019, the US FDA granted accelerated approval for Oxbryta™ (voxelotor) tablets for the…

Jim Gallagher2021 年 7 月 14 日

罕见病和孤儿病

利用一种罕见病的临床数据支持另一种罕见病的药物审批

A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease

Dose Optimization Using Population PK for an Orphan Drug

罕见病管理的“可怕”未来

Using a PBPK-PD Receptor Occupancy Model to Determine the Optimal Dose of Radiprodil for Neonates

Simcyp’s Age-Defining Feature Facilitates Everolimus Dosing in Neonates

Eliglustat for Gaucher Disease: Quantifying the Impact of Pharmacogenetic Status on DDIs

Using PBPK for Label Recommendations in Rare Disease: Deflazacort for Duchenne Muscular Dystrophy

使用 Simcyp 模拟器的 DDI 监管海报：Ibrutinib (Imbruvica®)

治疗镰状细胞病的 Voxelotor：无需任何临床研究，利用剂量预测模型进行 DDI 预测

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