Industry Insights

Industry Insights

Cell and gene therapies: Where are we now?

By CONTRIBUTOR

31 July 2024

RARE News

Our Voice, Our Rights, Our Future: The ALLIANCE report and video explores key themes and feedback from its 2024 annual conference

By admin

31 July 2024

RARE News

Innov4-ePiK: Innovative diagnostic and therapeutic approaches in potassium channel developmental and epileptic encephalopathies (K-DEEs) using 4P for medicine

By admin

29 July 2024

RARE News

cTAP Study indicates Duchenne muscular dystrophy clinical trial enrollment criteria should expand beyond ambulatory status

By admin

29 July 2024

RARE News

Landmarks shine pink and purple for National Eosinophilic Week

By admin

29 July 2024

Letters

Sarcoma Awareness Month: Sarcomas demand urgent awareness and education to prevent poor outcomes

By CONTRIBUTOR

24 July 2024

Turning the tide for rare disease

I am Celine Dion: The stiff person syndrome community reacts to the release of Dion’s documentary

By CONTRIBUTOR

22 July 2024

RARE News

The CMT Research Foundation’s 2024 Global CMT Research Convention to gather renowned scientific experts and patients to discuss status of treatments and cures for Charcot-Marie-Tooth (CMT) disease

By admin

19 July 2024

RARE News

NHL All-Star Ryan Getzlaf returns to the links for the 13th Annual Getzlaf Golf Shootout on September 13-14, benefiting CureDuchenne

By admin

17 July 2024

Industry Insights

Early access to medicines: A picture is worth a thousand words

By CONTRIBUTOR

17 July 2024

RARE News

Challenges and priorities for the PSP and CBD community: Insights from Mark Jackson

By admin

12 July 2024

RARE News

The CMT Research Foundation to launch grant program to support young investigators working on CMT

By admin

12 July 2024

Charity & advocacy

Bridging gaps in care: How independent charitable patient assistance organisations support underserved populations

By CONTRIBUTOR

10 July 2024

RARE News

Concerned stakeholders issue call to action on the implementation of the EU Joint Clinical Assessment for ATMPs

By admin

9 July 2024

Industry Insights

What can rare disease services learn from oncology services? A personal reflection

By CONTRIBUTOR

1 July 2024

RARE News

International Neonatal Screening Day: A call for global implementation of neonatal screening programmes

By admin

28 June 2024

RARE News

PSPA event brings together families affected by progressive supranuclear palsy and corticobasal degeneration

By admin

25 June 2024

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