Immunotherapeutics Emerge as Promising Treatments for MDS
Novel immunotherapeutic interventions have shown promise in harnessing the immune system’s capabilities to target malignant cells.
MDS Features
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Advocates Pushing for Safer, More Accessible Air Travel for People With Disabilities
Rare disease advocacy groups are lobbying Congress to amend regulations to let people with disabilities remain in their wheelchairs during flights.
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As Pandemic Restrictions Continue to Ease, Rare Disease Patients Still Fear COVID-19
Three years after COVID-19 was declared a pandemic, rare disease patients still fear the contagion—and must deal with the fallout.
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At 40, Orphan Drug Act Enjoys Rare Bipartisan Support
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
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Ambitious NCATS Bespoke Gene Therapy Consortium Targets Ultrarare Diseases
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
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EveryLife Foundation Navigates Complex Issues to Advocate for Rare Disease Patients
The EveryLife Foundation for Rare Diseases pushes a legislative agenda aimed at benefitting the 30 million Americans with a rare disease.
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