MPS Serbia

18/03/2026

Rezervišite svoje mesto na vreme

To ensure that as many of our colleagues, researchers, and advocates as possible can join us at the best possible rate, we are pleased to announce that the Early Bird registration deadline has been officially extended to March 15, 2026.

This extension is our way of supporting the diverse global community that makes this Symposium so unique. Take advantage of these additional weeks to finalize your plans and join us for this historic 20th-anniversary celebration at a reduced rate.

⏳ New Deadline: March 31, 2026
🔗 Register now to benefit from discounted rates: https://mps2026.com/

15/10/2025

The International MPS Network (IMPSN) would like to share an important update from Denali Therapeutics regarding the review of their investigational therapy for Hunter syndrome (MPS II).

On October 13, 2025, Denali announced that the U.S. Food and Drug Administration (FDA) has extended its review timeline for the Biologics License Application (BLA) seeking accelerated approval of tividenofusp alfa (DNL310).

🔹 New FDA target decision date: April 5, 2026 (previously January 5, 2026)
🔹 The extension follows Denali’s submission of additional clinical pharmacology information as part of the standard review process.
🔹 This update is not related to efficacy, safety, or biomarkers, and no new data were requested by the FDA.

We know this news brings disappointment and frustration for many families who have been waiting and hoping for new treatment options. The IMPSN shares in that sentiment and recognizes how deeply these delays can affect those living with MPS II and their loved ones.

At the same time, we remain hopeful. Denali continues to work closely with the FDA and has reaffirmed its commitment to advancing tividenofusp alfa responsibly and as quickly as possible.

The IMPSN will continue to advocate, communicate, and stand alongside every family as this process continues. Your resilience, unity, and strength continue to inspire all of us.

📄 Read Denali’s full announcement: https://investors.denalitherapeutics.com/news-releases/news-release-details/denali-therapeutics-announces-fda-review-extension-bla

08/07/2025

A major step forward for the MPS II (Hunter syndrome) community.

Denali Therapeutics has announced that the FDA has accepted its Biologics License Application (BLA) for tividenofusp alfa (DNL310) and granted it Priority Review, with a target action date of January 5, 2026.

This submission is supported by clinical trial data and reflects years of dedication from families, advocates, and researchers working to bring hope to those affected by Hunter syndrome. Denali is also continuing its global Phase 2/3 COMPASS study, advancing efforts beyond the U.S.

The IMPSN welcomes this encouraging progress and stands with the MPS II community as we await next steps in the FDA’s review.

📅 PDUFA Target Date: January 5, 2026
📰 Read the full release here:
👉 https://investors.denalitherapeutics.com/news-releases/news-release-details/denali-therapeutics-announces-fda-acceptance-and-priority-review

#​MPSCommunity

07/07/2025

🚨 BREAKING NEWS 🚨

We’re encouraged to share an important update for our ( ) community:

The FDA has accepted Denali Therapeutics' application for tividenofusp alfa (DNL310), a potential treatment for Hunter syndrome (MPS II). Even more encouraging—the FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of January 5, 2026

We will keep you updated as the process continues! In the mean time, you can read the full announcement here: https://ow.ly/JQsO50WlGSr

15/05/2025

29/02/2024

Podržavamo i nastavljamo svoje živote najbolje što možemo, sa osmehom na licu i snagom u grudima

15/02/2024

Save the date: 17th International Symposium on MPS and Related Diseases. Welcome!😀

30/01/2024

🚨THE RARE DISEASE DAY CHALLENGE IS BACK🚨

We are delighted to announce the return of our popular challenge from last year! 🥳 Throughout February, up to Rare Disease Day!

Each day participants will partake in a different Rare Disease Day themed challenge! All of the best ones will be shared on our social media!😁

Get ready to make every day count! Mark your calendars and join the Rare Disease Day Challenge from 1 to 29 February. Together, let's turn awareness into action and inspire change! 🌍💙

30/01/2024

Povodom usvojenog programa mera podrške za obolele od retkih bolesti, obaveštavamo vas da će sve informacije, u vezi same prijave i celokupnog procesa, biti objavljene u ponedeljk, 29. januara, na sajtu Ministarstva za brigu o porodici i demografiju.