Rare diseases Archives -

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that

Orphan drugs’ financial success raises questions

https://pharmaphorum.com/market-access-2/orphan-drugs-financial-success-raises-questions/

Events/ Events/ R&D

2nd Integrin-Targeted Drug Development Summit

Erin Thomas autoimmune, integrin, ophthalmology, Rare diseases 0 Comment

In collaboration with leading integrin drug developers, including Genentech, 7 Hills Pharma, and Pfizer, the 2nd Integrin-Targeted Drug Development Summit is designed for

2nd Integrin-Targeted Drug Development Summit

https://pharmaphorum.com/events/2nd-integrin-targeted-drug-development-summit/

News/ News/ Patients

Rare diseases have tenfold higher impact on healthcare costs; study

Phil Taylor Chiesi, IQVIA, Rare Disease Day, Rare diseases 0 Comment

A new study has suggested that the economic burden posed by rare diseases is around 10 times higher

Rare diseases have tenfold higher impact on healthcare costs; study

https://pharmaphorum.com/news/rare-diseases-have-tenfold-higher-impact-on-healthcare-costs-study/

R&D/ Views & Analysis/ Views and analysis

Rare Disease R&D: one step forward, but a long road ahead

Ben Hargreaves Rare Disease Day, rare disease R&D, Rare diseases 0 Comment

With over 40% of all medicines in the pipeline aimed at rare diseases, the future looks positive for

Rare Disease R&D: one step forward, but a long road ahead

https://pharmaphorum.com/r-d/rare-disease-rd-one-step-forward-but-a-long-road-ahead/

Market Access/ News/ News

Orphazyme ‘looking at options’ after lead drug hits buffers in EU

Phil Taylor Niemann-Pick type C, Orphazyme, Rare diseases 0 Comment

Orphazyme’s hopes of getting EU approval for its Niemann-Pick disease type C (NPC) therapy arimoclomol after a rejection

Orphazyme ‘looking at options’ after lead drug hits buffers in EU

https://pharmaphorum.com/news/orphazyme-looking-at-options-after-lead-drug-hits-buffers-in-eu/

Digital/ Events/ Events

Real-World Evidence 2022: Rare Diseases and Innovative Therapies

Darcy Hodge digital health, patient centricity, pharma, Rare diseases, Real World Evidence 0 Comment

RWE Live convenes those involved in RWE, HTA and more.

Real-World Evidence 2022: Rare Diseases and Innovative Therapies

https://pharmaphorum.com/events/real-world-evidence-2022-rare-diseases-and-innovative-therapies/

Copy of Bella Mitochondria-Targeted Drug Development - LinkedIn (2)

Events/ Events/ R&D

2nd Mitochondria-Targeted Drug Development Summit

jenna.warren@hansonwade.com drug development, Mitochondria, Mitochondrial Dysfunction, neurodegenerative, Rare diseases 0 Comment

Therapies for mitochondrial dysfunction

2nd Mitochondria-Targeted Drug Development Summit

https://pharmaphorum.com/events/2nd-mitochondria-targeted-drug-development-summit/

R&D/ Views & Analysis/ Views and analysis/ Webinars/ Webinars in home page/ Webinars On demand

The power of R&D collaborations to improve outcomes in rare diseases

mike.hammerton@pharmaphorum.com genomic medicine, IQVIA, orphan drugs, precision medicine, Rare diseases 0 Comment

Far more common than initially thought, rare disease treatments remain far too scarce. The answer to the problem,

The power of R&D collaborations to improve outcomes in rare diseases

https://pharmaphorum.com/webinars/pharma-research-outcomes-rare-diseases/

Sales and Marketing/ Views & Analysis/ Views and analysis

The benefits of being a family-run pharma company

mike.hammerton@pharmaphorum.com Chiesi, Rare diseases 0 Comment

The third generation of his family to hold a leadership role at Chiesi Group, Giacomo Chiesi reflects on

The benefits of being a family-run pharma company

https://pharmaphorum.com/sales-marketing/chiesi-family-run-pharma-company-benefits/

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