House Approves Extending Vouchers for Pediatric Rare Disease …
Rare disease advocacy groups praised the House’s passage of legislation that extends the Rare Pediatric Disease Priority Review Voucher program until 2029.
ANCA-Associated Vasculitis (AAV) - Rare Disease Advisor
Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a heterogeneous group of rare autoimmune diseases in which ANCA target and activate neutrophils. These, in turn, …
Rare Disease News, Perspectives & Resources - Rare Disease Advisor
Rare Disease News is the premier online resource for the latest rare disease news, perspectives and information.
Chronic Lymphocytic Leukemia Types - Rare Disease Advisor
Chronic lymphocytic leukemia (CLL) is categorized as a mature B-cell neoplasm, under the subcategory of pre-neoplastic and neoplastic small lymphocytic
IgG4-Related Disease Life Expectancy - Rare Disease Advisor
Are you a health care provider looking for information on the life expectancy of immunoglobulin G4-related disease (IgG4-RD)? Read more here.
ANCA-Associated Vasculitis (AAV) - Rare Disease Advisor
Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) comprises a group of rare autoimmune conditions characterized by inflammation and the destruction of …
Spinal Muscular Atrophy Clinical Trials - Rare Disease Advisor
Are you a healthcare provider looking for information on spinal muscular atrophy (SMA) clinical trials? Read more here.
Achondroplasia Life Expectancy - Rare Disease Advisor
Oct 22, 2024 · Most individuals with achondroplasia, the most common type of short-limbed and short-stature dwarfism, have a normal or near-normal life expectancy.1 The
Achondroplasia History - Rare Disease Advisor
Achondroplasia is the most common skeletal dysplasia in humans that affects bone growth and results in disproportionate short stature. This condition
Rilzabrutinib Induces Rapid, Sustained Platelet Response in ITP
Dec 16, 2024 · Rilzabrutinib may be a promising and effective therapy for patients with primary immune thrombocytopenia (ITP) who are unresponsive or intolerant to standard therapies, …