NORWELL, Mass., Dec. 8, 2025 — The National Organization for Rare Disorders (NORD®), a leading rare disease patient advocacy organization, today announced the appointment of Kim Isenberg, a retired industry … Continue reading "Public Affairs and Patient Advocacy Leader Kim Isenberg Joins National Organization for Rare Disorders Board of Directors"

Original title: “Public Affairs and Patient Advocacy Leader Kim Isenberg Joins National Organization for Rare Disorders Board of Directors”

NORD report shows 63 FDA vouchers driving breakthroughs for 47 rare pediatric diseases, 43 with no prior treatments — unprecedented progress for children left behind WASHINGTON, Dec. 4, 2025 — … Continue reading "America’s Rare Children Need Congress to Act: NORD Urges Swift Reauthorization of Proven Rare Pediatric Disease Voucher Program"

Original title: “America’s Rare Children Need Congress to Act: NORD Urges Swift Reauthorization of Proven Rare Pediatric Disease Voucher Program”

The post Journée Annuelle de l’AIRG – France appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Journée Annuelle de l’AIRG – France”

NORD’s growing network, now 46 institutions strong, represents a coordinated nationwide effort to strengthen rare disease care and research collaboration NORWELL, Mass., Dec. 3, 2025 — The National Organization for … Continue reading "National Organization for Rare Disorders Adds Seven Rare Disease Centers of Excellence Nationwide"

Original title: “National Organization for Rare Disorders Adds Seven Rare Disease Centers of Excellence Nationwide”

In this NORD Guest Blog, two NORD Member patient organizations, TSC Alliance & Dup15q Alliance, discuss the importance of infantile spasms awareness, the symptoms to look out for, and how … Continue reading "Guest Blog: How Collaboration Drives Early Diagnosis and Better Outcomes During Infantile Spasms Awareness Week"

Original title: “Guest Blog: How Collaboration Drives Early Diagnosis and Better Outcomes During Infantile Spasms Awareness Week”

The post AFM-Téléthon 2025 appeared first on EURORDIS-Rare Diseases Europe.

Original title: “AFM-Téléthon 2025”

The post ERN ReCONNET: Sharing Good Clinical Practice 2025 Event appeared first on EURORDIS-Rare Diseases Europe.

Original title: “ERN ReCONNET: Sharing Good Clinical Practice 2025 Event”

The post Patient Partnership Webinar: The UnDiagnosed Patients Working Group by ERN LUNG appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Patient Partnership Webinar: The UnDiagnosed Patients Working Group by ERN LUNG”

Updated on 27 November 2025: To extend the deadline for submissions of applications from 23 November to 5 December 2025 , The European Commission has launched a call for expressions of interest to represent patient and healthcare professional organisations in EMA’s Paediatric Committee (PDCO).…

Original title: “Call for expressions of interest for civil society representatives to participate in the work of EMA’s Paediatric Committee”

The post HLM RARE appeared first on EURORDIS-Rare Diseases Europe.

Original title: “HLM RARE”

The post European Conference on Rare Diseases and Orphan Products 2026 appeared first on EURORDIS-Rare Diseases Europe.

Original title: “European Conference on Rare Diseases and Orphan Products 2026”

Implementation of two new patient registries on the IAMRARE® platform funded by RDCA-DAP® DANBURY, Conn., Nov. 14, 2025 — The National Organization for Rare Disorders (NORD®), a leading national nonprofit … Continue reading "NORD Launches New RFP for Patient Registries"

Original title: “NORD Launches New RFP for Patient Registries”

For one powerful day in Milwaukee, more than 110 people impacted by rare diseases — patients, caregivers, families, clinicians, and advocates — came together to share something simple yet profound: … Continue reading "Living Rare, Living Stronger: A Day of Connection, Courage, and Community in Milwaukee"

Original title: “Living Rare, Living Stronger: A Day of Connection, Courage, and Community in Milwaukee”

11 people from 7 states have gotten sick from the same strain of Salmonella.

Original title: “Salmonella outbreak linked to moringa leaf, an ingredient in powdered supplements”

CDC urges people to check their refrigerators and freezers for recalled foods. Do not eat them.

Original title: “CDC continues to note illnesses despite product recalls and asks consumers to check for recalled foods in <em>Listeria</em> outbreak linked to prepared meals”

Our latest ERTC workshop explored integrating patient experience data into medicines regulation. The post Twenty years of dialogue: EURORDIS Round Table of Companies marks its 40th workshop appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Twenty years of dialogue: EURORDIS Round Table of Companies marks its 40th workshop”

This session recording is from Oct.  16, 2025  Rare Advocacy Exchange Session 8: Knowing the Rights for […] The post RARE Advocacy Exchange Session 8, Knowing Your Rare Rights appeared first on Global Genes.

Original title: “RARE Advocacy Exchange Session 8, Knowing Your Rare Rights”

We identify priorities for the Biotech Act to ensure it delivers real progress for our community. The post Why the EU Biotech Act will – and how it must – matter for rare diseases appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Why the EU Biotech Act will – and how it must – matter for rare diseases”

This Global Genes Quick Guide is a resource for advocates focused on pressing topics causing challenges in […] The post Global Genes Guide to Starting a Nonprofit Patient Advocacy Organization appeared first on Global Genes.

Original title: “Global Genes Guide to Starting a Nonprofit Patient Advocacy Organization”

This session recording is from Sept.  18, 2025  Rare Advocacy Exchange Session 7: Getting A Precise Diagnosis […] The post RARE Advocacy Exchange Session 7, Getting a Precise Genetic Diagnosis appeared first on Global Genes.

Original title: “RARE Advocacy Exchange Session 7, Getting a Precise Genetic Diagnosis”