Rare Disease

News

Novartis buys Pharming priority review voucher for a song

Pharming has agreed a deal to sell a recently awarded priority review voucher (PRV) to Novartis for around $21 million - well below the market rate.

R&D

Health Innovators – Jennifer Schranz

In the rare and ultra-rare disease space, the paucity of both data and patients makes everything about research and development more complicated.

Digital

Combatting longstanding challenges in rare disease detection...

Approximately 350 million people are living with up to 8,000 rare diseases worldwide.

News

FDA clears Vyjuvek as first topical gene therapy

The FDA has approved Krystal Biotech’s gene therapy Vyjuvek for dystrophic epidermolysis bullosa (DEB), a rare genetic disease characterised by fragile skin that can split

R&D

A closer look at gene therapy with PTC Therapeutics

In the latest instalment of the pharmaphorum podcast, editor-in-chief Jonah Comstock connects with Kylie O’Keefe to talk about gene therapy in rare diseases.

News

PTC eyes filings for PKU therapy after phase 3 win

PTC Therapeutics is on track to file for approval of a new therapy for rare disease phenylketonuria after a positive phase 3 readout that could extend its product portfoli

Novartis buys Pharming priority review voucher for a song

Health Innovators – Jennifer Schranz

Combatting longstanding challenges in rare disease detection...

FDA clears Vyjuvek as first topical gene therapy

A closer look at gene therapy with PTC Therapeutics

PTC eyes filings for PKU therapy after phase 3 win

GSK sets up Nucala and Dupixent showdown in COPD

Health Trends 2025: Reimagining What's Possible

Others Old
-About pharmaphorum
-Articles
--eyeforpharma Barcelona 2015
-Audio
-barelonalive 2016
-Cancer
-Diabetes
--Diabetes
-Disease Spotlight
--DMD Main
--LHON Main
-ESMO2016
-ESMO2016-related
-Event coverage
--ASCO-slider
--ASCO-pp
--ASCO-contributor
--ASCO 2018
-Featured content right column
-Featured Event
-Jobs
--pharmaphorum-live
-Medical Affairs Spotlight
--Medical Affairs Spotlight - partner
--Medical Affairs Spotlight - main
-Podcast
-Premium media
-Quintiles
--Quintiles-oncol-PC
--Quintiles - ContentIO
--Quintiles - Articles
--Quintiles - Oncol articles
-Roundtables
--AZ-roundtable-biographies-2017
--AZ-roundtable-interviews-2017
--AZ-roundtable-debate-2017
-Sales and Marketing
--News
--Insights
--Appointments
--Debates
--Debates & Insight
--Views & Analysis
--Partner Content
-Slider in events
-Slider in home page
-Slider in video
-Slider in views and analysis
-Slider in webinars
-Slider in whitepapers
-Spotlight
--Understanding HCPs
--Future of Generics
--Frontiers Health 2022
--Frontiers Health 2021
--Behavioural Science
--ASCO 2019 Live coverage
--AI in Pharma Marketing
--ASCO 2019 and the future of oncology
-Startups
--Views & Analysis
--News
-System
-Top pharmaceutical companies
--Bristol-Myers Squibb
--Teva
--Roche
--Pfizer
--Novo Nordisk
--Novartis
--Merck Inc
--GlaxoSmithKline
--Gilead
--AstraZeneca
--AbbVie
-Top pharmaceutical companies
--Merck Inc
-Top stories
-Transversal Categories
--Brexit
--Clinical
--Digital and Social Media
--US
---Articles
--UK & Europe
--Sales and Marketing
--Rare diseases
--R&D
--Pharma Market Access
--M&A
--Healthcare
-Veeva
-Veeva channel content articles
-Veeva channel content webinars
-Video
-Views and analysis
-Webinars in home page
-Whitepapers
Others
-Transversal Categories
--Sustainability
--Clinical
--Brexit
--Digital and Social Media
--Healthcare
--US
--UK & Europe
--Sales & Marketing
--Rare diseases
--R&D
--Pharma Market Access
--M&A
-Top stories
-About pharmaphorum
-Top pharmaceutical companies
--Gilead
--Merck Inc
--Teva
--Roche
--Pfizer
--Novo Nordisk
--Novartis
--GlaxoSmithKline
--Bristol-Myers Squibb
--AstraZeneca
--AbbVie
-test webinar
-System
-Startups
--News
--Views & Analysis
-Veeva channel content webinars
-Webinars in home page
--Live webinar
--Webinars On demand
-Veeva channel content articles
-Veeva
-Spotlight
-Event coverage
--ASCO-slider
--ASCO-pp
--ASCO-contributor
--ASCO 2018
-ESMO2016-related
-ESMO2016
-Featured Event
-Disease Spotlight
--LHON Main
--DMD Main
-Diabetes
--Diabetes
-Cancer
-barelonalive 2016
-Audio
-Articles
--eyeforpharma Barcelona 2015
-Slider in whitepapers
-Featured content right column
-Jobs
--pharmaphorum-live
-Roundtables
--AZ-roundtable-interviews-2017
--AZ-roundtable-biographies-2017
--AZ-roundtable-debate-2017
-Slider in webinars
-Slider in views and analysis
-Slider in video
-Slider in home page
-Slider in events
-Quintiles
--Quintiles-oncol-PC
--Quintiles - Oncol articles
--Quintiles - ContentIO
--Quintiles - Articles
-Premium media
-Medical Affairs Spotlight
--Medical Affairs Spotlight - partner
--Medical Affairs Spotlight - main
Home
-News
-Views & Analysis
-Deep Dive
-Webinars
--Live webinar
-Podcasts
-Videos
-White Papers
-Events
--JP Morgan
--LSX Investech
--Anthropy
--Reuters Pharma 2022
--World Cancer Series
--Hlth 2022
--PM Society Awards
--Patient Summit PC
--Frontiers Health 2022
--FHlive17
--ASCO 2018
-Digital Debates
-Podcasts & Videos
-Partner Content
--Events
--News
--Appointments
--Press releases
Digital
-Appointments
-Debates
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
Market Access
-Debates
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
Oncology
-Appointments
-Debates
-Insights
-News
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
Patients
-Debates
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
R&D
-Appointments
-Debates
-Debates & Insights
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Partner Content
-Debates & Insight
Sales & Marketing
-Debates
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
Spotlight on
-AI in Pharma Marketing
-ASCO 2019 and the future of oncology
-ASCO 2019 Live coverage
-Behavioural Science
-Frontiers Health 2021
-Frontiers Health 2022
-Frontiers Health 2023
-Frontiers Health 2024
-Future of Generics
-Life Sciences Industry Report
-Understanding HCPs
-World Without Disease Summit
-The Future of Generics

Others Old
-About pharmaphorum
-Articles
--eyeforpharma Barcelona 2015
-Audio
-barelonalive 2016
-Cancer
-Diabetes
--Diabetes
-Disease Spotlight
--DMD Main
--LHON Main
-ESMO2016
-ESMO2016-related
-Event coverage
--ASCO-slider
--ASCO-pp
--ASCO-contributor
--ASCO 2018
-Featured content right column
-Featured Event
-Jobs
--pharmaphorum-live
-Medical Affairs Spotlight
--Medical Affairs Spotlight - partner
--Medical Affairs Spotlight - main
-Podcast
-Premium media
-Quintiles
--Quintiles-oncol-PC
--Quintiles - ContentIO
--Quintiles - Articles
--Quintiles - Oncol articles
-Roundtables
--AZ-roundtable-biographies-2017
--AZ-roundtable-interviews-2017
--AZ-roundtable-debate-2017
-Sales and Marketing
--News
--Insights
--Appointments
--Debates
--Debates & Insight
--Views & Analysis
--Partner Content
-Slider in events
-Slider in home page
-Slider in video
-Slider in views and analysis
-Slider in webinars
-Slider in whitepapers
-Spotlight
--Understanding HCPs
--Future of Generics
--Frontiers Health 2022
--Frontiers Health 2021
--Behavioural Science
--ASCO 2019 Live coverage
--AI in Pharma Marketing
--ASCO 2019 and the future of oncology
-Startups
--Views & Analysis
--News
-System
-Top pharmaceutical companies
--Bristol-Myers Squibb
--Teva
--Roche
--Pfizer
--Novo Nordisk
--Novartis
--Merck Inc
--GlaxoSmithKline
--Gilead
--AstraZeneca
--AbbVie
-Top pharmaceutical companies
--Merck Inc
-Top stories
-Transversal Categories
--Brexit
--Clinical
--Digital and Social Media
--US
---Articles
--UK & Europe
--Sales and Marketing
--Rare diseases
--R&D
--Pharma Market Access
--M&A
--Healthcare
-Veeva
-Veeva channel content articles
-Veeva channel content webinars
-Video
-Views and analysis
-Webinars in home page
-Whitepapers
Others
-Transversal Categories
--Sustainability
--Clinical
--Brexit
--Digital and Social Media
--Healthcare
--US
--UK & Europe
--Sales & Marketing
--Rare diseases
--R&D
--Pharma Market Access
--M&A
-Top stories
-About pharmaphorum
-Top pharmaceutical companies
--Gilead
--Merck Inc
--Teva
--Roche
--Pfizer
--Novo Nordisk
--Novartis
--GlaxoSmithKline
--Bristol-Myers Squibb
--AstraZeneca
--AbbVie
-test webinar
-System
-Startups
--News
--Views & Analysis
-Veeva channel content webinars
-Webinars in home page
--Live webinar
--Webinars On demand
-Veeva channel content articles
-Veeva
-Spotlight
-Event coverage
--ASCO-slider
--ASCO-pp
--ASCO-contributor
--ASCO 2018
-ESMO2016-related
-ESMO2016
-Featured Event
-Disease Spotlight
--LHON Main
--DMD Main
-Diabetes
--Diabetes
-Cancer
-barelonalive 2016
-Audio
-Articles
--eyeforpharma Barcelona 2015
-Slider in whitepapers
-Featured content right column
-Jobs
--pharmaphorum-live
-Roundtables
--AZ-roundtable-interviews-2017
--AZ-roundtable-biographies-2017
--AZ-roundtable-debate-2017
-Slider in webinars
-Slider in views and analysis
-Slider in video
-Slider in home page
-Slider in events
-Quintiles
--Quintiles-oncol-PC
--Quintiles - Oncol articles
--Quintiles - ContentIO
--Quintiles - Articles
-Premium media
-Medical Affairs Spotlight
--Medical Affairs Spotlight - partner
--Medical Affairs Spotlight - main
Home
-News
-Views & Analysis
-Deep Dive
-Webinars
--Live webinar
-Podcasts
-Videos
-White Papers
-Events
--JP Morgan
--LSX Investech
--Anthropy
--Reuters Pharma 2022
--World Cancer Series
--Hlth 2022
--PM Society Awards
--Patient Summit PC
--Frontiers Health 2022
--FHlive17
--ASCO 2018
-Digital Debates
-Podcasts & Videos
-Partner Content
--Events
--News
--Appointments
--Press releases
Digital
-Appointments
-Debates
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
Market Access
-Debates
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
Oncology
-Appointments
-Debates
-Insights
-News
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
Patients
-Debates
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
R&D
-Appointments
-Debates
-Debates & Insights
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Partner Content
-Debates & Insight
Sales & Marketing
-Debates
-Insights
-News
-Press Releases
-Views & Analysis
-Deep Dive
-Webinars
-Podcasts
-Video
-White Papers
-Event
-Debates & Insight
-Partner Content
Spotlight on
-AI in Pharma Marketing
-ASCO 2019 and the future of oncology
-ASCO 2019 Live coverage
-Behavioural Science
-Frontiers Health 2021
-Frontiers Health 2022
-Frontiers Health 2023
-Frontiers Health 2024
-Future of Generics
-Life Sciences Industry Report
-Understanding HCPs
-World Without Disease Summit
-The Future of Generics

Rare Disease

Editor's Picks