Dipanjan Basu, PhD, Highlights Potential of Omipalisib for Neurocutaneous Melanocytosis
Dipanjan Basu, PhD, is testing non-melanoma drugs, like the investigational therapy omipalisib (GSK2126458) in cultured patient cells.
Top 5 Rare Disease News of the Week—October 14, 2018
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
Genetic Testing for Rare Diseases with Geneticist, Debra Regier, MD
In a recent webinar, geneticist Debra Regier, MD, provides an overview of the importance of genetic testing in the rare disease space.
FDA Grants Fast Track Designation to Betalutin for Follicular Lymphoma
e FDA grants Fast Track designation to 177Lu-lilotomab satetraxetan (Betalutin) for the treatment of patients with relapsed/refractory follicular lymphoma after at least 2 prior systemic therapies.
"Right to Try" Bill Heads to President Trump for Approval
Congress has voted to pass the Right to Try bill which will provide terminally ill patients with the ability to seek out experimental therapies not yet approved by the FDA.
NORD: The Voice of the Community - May 2018
NORD's Voice of the Community - May 2018
NORD to Celebrate 2018 Rare Impact Awards and 35th Anniversary
On May 17th, the National Organization of Rare Disorders (NORD) will host its 2018 Rare Impact Awards in Washington, DC at the Andrew W. Mellon Auditorium.
NORD Director of Federal Policy Emphasizes Patient Engagement
Paul Melmeyer, Director of Federal Policy for the National Organization for Rare Disorders (NORD) wants to do his part to ensure that the proper choices are being made on behalf of people affected by rare diseases.
FDA Observes Rare Disease Day with Help from NORD, NIH
In observance of World Rare Disease Day, the U.S. FDA has announced a collaboration with NORD, in which a pilot project would assist medical reviewers in better understanding rare disease patient experiences.
NORD: The Voice of the Community - February 2018
NORD's Voice of the Community - February 2018
Q&A with the Director of the Children's National Rare Disease Institute
Marshall Summar, M.D., director of the Children’s National Rare Disease Institute, sat down with Rare Disease Report to discuss recent news and what it means for the rare disease community.
Rare Disease Day 2018 Starts with 7,000 Mile Trek
NORD, the leading nonprofit organization dedicated to helping Americans with rare diseases, announced this morning that its 7,000 Mile Rare Movement will be kicking off this year’s celebration.
NORD: The Voice of the Community - January 2018
NORD's Voice of the Community - January 2018
Retrophin Donates $3 Million to Create Rare Disease Network
Retrophin has committed $3 million over the next 6 years to support the work of the Children’s National Rare Disease Institute, the first Center of Excellence for the National Organization for Rare Disorders.
New Process Yields Better Insulin
The quest for manufactured insulin that mirrors the real thing has progressed a step forward.
Lipoprotein(a) and coronary artery disease:
The well-established causative role of low-density lipoprotein particles in atherosclerotic plaque development has led to this biomarker being a primary target of treatment in the prevention of coronary artery disease.
