RDR Alert®

Between 2006-2016, the hospital mortality rates for patients with ILD did not change.

A discussion on unanswered questions and concerns among clinicians related to sickle cell disease and the ongoing COVID-19 pandemic.

The injection therapy has shown capability to stop or even reverse the progression of early puberty in patients with the rare disease.

The Chief Medical Officer of the Sickle Cell Disease Association of America discusses a recently released care provider advisory from the organization.

The subcuntaenous therapy's base drug was approval for two separate multiple myeloma indications last year.

Results of the study from investigators at CHOP could have real-world impact on treatment algorithms.

ABG-023 overcomes the limitations glucagon because it remains stable in solutions.

This marks ibrutinib's 11th FDA approval.

Pathogenic mechanism between respiratory exposures and the development of AAV could be established in future research.

The drug is the first of its kind to be indicated for the genetic nervous disorder.

Study findings show the treatment is safe and effective at stopping bleeding episodes in patients with hemophilia A or B.

APR-OD031 will be taken in combination with a patented drug delivery system to treat the rare inherited disease.

In the phase 2 PIONEER trial, patients taking avapritinib self-reported improvements in quality of life.

Osilodrostat oral tablets are indicated for the treatment of Cushing's disease in patients not eligible for surgery or in those who still have surgery despite the disease.

Approval of isatuximab-irfc (Sarclisa) was based on the results of a 307-person trial, according to a statement from the FDA.

Sika Dunyoh, director of education programs with NORD, discusses that year in rare disease and what to expect moving forward.

The RareWear program is designed to bridge medical device companies to rare disease patient applicants, seeking better disease and treatment monitoring.

Advanced cystic fibrosis liver disease manifests as portal hypertension with or without cirrhosis in about 7% of patients with cystic fibrosis.

New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.

An emphasis on collecting and assessing quality-of-life biomarkers shows promise for tailoring treatment of the rare condition.

What is the primary care physician's role in detecting the rare hepatologic condition?

Inovoio Pharmaceuticals will assess INO-3107 to eventually become the first drug indicated for the rare disease.

The IGF-1R inhibitor becomes the first therapy indicated for the rare eye condition.

Avapritinib represents the first ever treatment for a rare mutation for patients with gastrointestinal stromal tumors.

The new indication will allow the longtime ALS therapy to be used among the 85% of patients required to use PEG due to dysphagia.