Rare Disease Report&reg Editorial Staff

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

In a recent interview with Rare Disease Report®, Hartmann Wellhoefer, MD, discussed the biggest challenges faced and advances being made in the fight against MPS.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.

The FDA has approved a device intended for the treatment of acute coronary heart perforation, a rare, but potentially life-threatening complication of heart vessel procedures.

The FDA has approved the subcutaneous formulation of tocilizumab (Actemra) for the treatment of active systemic juvenile idiopathic arthritis in patients two years of age and older.

Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.

Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.

The FDA has approved ibrutinib (IMBRUVICA) plus rituximab (RITUXAN) for the treatment of adult patients with Waldenström's macroglobulinemia, a rare and incurable type of non-Hodgkin's lymphoma.

Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.

The FSA approves lanadelumab (Takhzyro), the first monoclonal antibody approved in the United States to treat patients 12 years and older with types I and II hereditary angioedema.

The FDA has approved stiripentol (Diacomit) for the treatment of seizures associated with Dravet syndrome, a rare form of epilepsy.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

Pauline Funchain MD, explains how better understanding of genetics can help shape future treatments for rare cancers.

The FDA has approved lenvatinib capsules (Lenvima, Eisai Inc.) for first-line treatment of patients with unresectable hepatocellular carcinoma.

The FDA has approved ivacaftor (KALYDECO) as the first and only medicine to treat the underlying cause of cystic fibrosis in children ages 12 to

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

The FDA has approved mogamulizumab-kpkc (Poteligeo) injection for intravenous use for the treatment of adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome after at least 1 prior systemic therapy.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

Manmeet Ahluwalia, MD, discusses the challenges patients and providers face when it comes to rare cancers.

The FDA has approved iobenguane I 131 (Azedra) injection for intravenous use for the treatment of adults and adolescents 12 years of age and older with rare adrenal gland tumors such as pheochromocytoma or paraganglioma that cannot be surgically removed, have spread beyond the original tumor site, and require systemic anticancer therapy.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

E. Anders Kolb, MD, outlines the challenges in diagnosing pediatric cancers and explains how molecular events define disease outcomes.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

The FDA has approved ivosidenib tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia who have a specific genetic mutation.

E. Anders Kolb, MD, emphasizes the difference between pediatric and adult cancers and the need to develop new treatments developed specifically for children.

Pauline Funchain, MD, discusses the use of multiplex germline testing for rare diseases and cancers and explains the benefits of uncovering genetic factors.

Owen A. O’Connor, MD, PhD, outlines the importance of clinical trials and reviewed strides made in lymphoma treatments.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.