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2 articles matching "rare disease policy"

AdvocacyRSSMar 26

NORD Advances Policy Leadership with Strategic Appointments to Strengthen Rare Disease Advocacy

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.
ℹ Good to knowRead →
Clinical trialCLINICALTRIALSMar 26

Trial Now Recruiting: Diagnosis and Treatment of Patients With Inborn Errors of Metabolism (NCT00369421)

Researchers at the National Institutes of Health are looking for up to 4,000 patients with rare metabolic disorders to study and treat. These are conditions where the body cannot properly break down certain substances because of missing or faulty proteins. Patients will receive exams and treatment, mostly as outpatients, though some may need to stay at the NIH Clinical Center for special tests.

WHY IT MATTERSThis trial offers patients with arterial calcification due to CD73 deficiency direct access to specialized NIH researchers and potential treatment options that may not be available elsewhere.
→ You can act on thisArterial Calcification Due to Deficiency of CD73Inborn Errors of MetabolismRead →

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