Orphan Drug Archives

Successful Approaches from Rare Disease and Cellular and Gene Therapy Product Approvals

February 28, 2023

Approvals News

MediWound’s NexoBrid is FDA Approved for the Treatment of Severe Thermal Burns

January 4, 2023

Gene Therapies and Rare Diseases: Advancing Research with Humanized Models and AI

December 09, 2022

Blogs

UK’s NHS Backs World’s Costliest Drug for Treatment of MLD + Report Finds Increase in Pharma Cybersecurity Threats – Xtalks Life Science Podcast Ep. 48

February 16, 2022

Approvals News

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

February 14, 2022

Rare Disease Drug Launch – Lessons Learned to Ensure Success is Not a Rare Event

October 09, 2020

Biotech Blogs

The Challenges of Bringing Orphan Drugs to Market

February 28, 2019

Clinical Trial News

Alkeus Pharmaceuticals and OncoImmune Among Recipients of FDA’s Rare Disease Clinical Trials Grants

September 26, 2018

Venerated and Vital: Using Lipid Based Delivery for Fast and Efficient Early Phase Development

October 16, 2018

Biotech Blogs

Five Untapped Rare Diseases for Orphan Drug Development

April 19, 2018

Easing the Rare Disease Patient’s Burden in Clinical Trials with Innovative Technology

May 08, 2018

Approvals News

First New Treatment in Two Decades for Sickle Cell Disease Approved by FDA

July 11, 2017

Orphan Drug

Key Strategies for Successful Orphan Drug Launch in Europe

Axsome Therapeutics’ Reboxetine Promises Long-Term Symptom Relief in Narcolepsy

Navigating the Complexities of Rare Disease — A Quest Diagnostics Perspective

Sohonos (Palovarotene) Sets Milestone as First Drug for Ultra-Rare Bone Disease

Successful Approaches from Rare Disease and Cellular and Gene Therapy Product Approvals

MediWound’s NexoBrid is FDA Approved for the Treatment of Severe Thermal Burns

Gene Therapies and Rare Diseases: Advancing Research with Humanized Models and AI

UK’s NHS Backs World’s Costliest Drug for Treatment of MLD + Report Finds Increase in Pharma Cybersecurity Threats – Xtalks Life Science Podcast Ep. 48

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

Rare Disease Drug Launch – Lessons Learned to Ensure Success is Not a Rare Event

The Challenges of Bringing Orphan Drugs to Market

Alkeus Pharmaceuticals and OncoImmune Among Recipients of FDA’s Rare Disease Clinical Trials Grants

Venerated and Vital: Using Lipid Based Delivery for Fast and Efficient Early Phase Development

Five Untapped Rare Diseases for Orphan Drug Development

Easing the Rare Disease Patient’s Burden in Clinical Trials with Innovative Technology

First New Treatment in Two Decades for Sickle Cell Disease Approved by FDA

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Key Strategies for Successful Orphan Drug Launch in Europe

Axsome Therapeutics’ Reboxetine Promises Long-Term Symptom Relief in Narcolepsy

Navigating the Complexities of Rare Disease — A Quest Diagnostics Perspective

Sohonos (Palovarotene) Sets Milestone as First Drug for Ultra-Rare Bone Disease

Successful Approaches from Rare Disease and Cellular and Gene Therapy Product Approvals

MediWound’s NexoBrid is FDA Approved for the Treatment of Severe Thermal Burns

Gene Therapies and Rare Diseases: Advancing Research with Humanized Models and AI

UK’s NHS Backs World’s Costliest Drug for Treatment of MLD + Report Finds Increase in Pharma Cybersecurity Threats – Xtalks Life Science Podcast Ep. 48

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

Rare Disease Drug Launch – Lessons Learned to Ensure Success is Not a Rare Event

The Challenges of Bringing Orphan Drugs to Market

Alkeus Pharmaceuticals and OncoImmune Among Recipients of FDA’s Rare Disease Clinical Trials Grants

Venerated and Vital: Using Lipid Based Delivery for Fast and Efficient Early Phase Development

Five Untapped Rare Diseases for Orphan Drug Development

Easing the Rare Disease Patient’s Burden in Clinical Trials with Innovative Technology

First New Treatment in Two Decades for Sickle Cell Disease Approved by FDA

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