Articles related to RARE CONDITIONS & DISEASES

Home-based therapies are emerging as a viable solution to the challenges faced by global healthcare systems, particularly for conditions like sickle cell disease. Dive deeper into the research and implications for patient care.

Explore the unmasking of postural orthostatic tachycardia syndrome (POTS) amid the COVID-19 pandemic and its vaccine era, with the integration of traditional and alternative treatments offering renewed hope for affected patients.

Gain insight into how a shift in cellular environment and UV radiation exposure can instigate additional mutations, deepening our understanding of the evolution and development of rare and complex malignancies such as BPDCN.

The Low-PV phase 2 trial provides compelling evidence for the use of ropeginterferon alfa-2b in the management of low-risk Polycythemia vera, demonstrating its superiority over the standard treatment of therapeutic phlebotomy and aspirin in maintaining hematocrit levels and reducing disease-related symptoms.

In this randomized trial, standard treatment of rifampin and isoniazid for 24 weeks plus pyrazinamide and ethambutol for the initial 8 weeks was compared with an initial treatment of bedaquiline–linezolid for 8 weeks, extended treatment for persistent clinical disease, monitoring after treatment, and retreatment for relapse. The shorter approach was determined to be non-inferior.

According to Aimee Tharaldson, PharmD, a senior clinical pharmacist who spoke at the AMCP Nexus 2022 meeting, specialty medications continue to lead FDA approvals, with nearly half being developed for rare diseases. In 2023, many candidates will seek an orphan drug indication, which is a special FDA designation that provides tax credits for qualified clinical trials, exemption from user fees, and up to seven years of market exclusivity after approval. An estimated 7,000 orphan diseases exist.