AdvocacyRSS3 days ago
A person with SMA type 2 shares how a children's pamphlet called 'Meet Connie' helped them learn to advocate for themselves starting in third grade. The article reflects on how this resource supported their development as they navigated life with a power wheelchair and a chronic condition. Self-advocacy skills learned early can help young people with rare diseases communicate their needs to doctors, teachers, and others.
WHY IT MATTERSFor families raising children with SMA type 2, this story demonstrates how age-appropriate educational materials can build confidence and self-advocacy skills that improve long-term health outcomes and quality of life.
AdvocacyRSSMay 1
May is ALS Awareness Month, when organizations around the world work to educate people about ALS (a disease that affects nerve cells that control muscles) and raise money to help patients get better care. The Muscular Dystrophy Association and other groups are hosting events and encouraging people to support the ALS community through donations and advocacy.
WHY IT MATTERSALS Awareness Month creates focused momentum for fundraising and advocacy that directly supports access to care, treatments, and research for people currently living with ALS.
AdvocacyPRESS RELEASEMar 26
The National Organization for Rare Disorders (NORD), a major patient advocacy group, announced new leadership positions focused on policy and government relations. These appointments aim to strengthen NORD's efforts to influence laws and policies that affect people with rare diseases. The moves show NORD is expanding its work to advocate for patients at the federal and global levels.
WHY IT MATTERSStronger policy leadership at NORD means patients with rare diseases will have more powerful advocates working directly with Congress and government agencies to push for faster drug approvals, better insurance coverage, and increased research funding.
AdvocacyRSSMar 26
The National Organization for Rare Disorders (NORD), a major patient advocacy group, announced new leadership positions to strengthen its work in rare disease policy. Michael J. Beard was appointed as Vice President of Federal and Global Public Affairs. These leadership changes are designed to help NORD better represent patients' interests in government and international discussions about rare diseases.
WHY IT MATTERSStronger NORD leadership in policy and advocacy can directly influence which rare diseases get research funding, how quickly new treatments are approved, and what insurance coverage looks like for rare disease patients.
AdvocacyPUBMEDMar 26
This study looked at how patient groups led by people with rare diseases help fill gaps in education and awareness in Poland. Researchers interviewed 11 leaders of these patient groups to understand how they act as 'ambassadors' and 'advocates' to teach doctors, teachers, and the public about rare diseases. The findings show that patient advocacy groups play an important but often overlooked role in helping people understand and navigate rare diseases.
WHY IT MATTERSIf you have a rare disease, this research validates that patient-led advocacy groups are essential resources for getting accurate information and support when healthcare systems and professionals lack rare disease knowledge.