Articles related to RARE CONDITIONS & DISEASES

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.

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.

The diagnosis and treatment of mantle cell lymphoma are covered in this podcast by Jonathon Cohen, MD, MS, Associate Professor, Hematology and Medical Oncology, Emory University School of Medicine. He also discusses how BTK inhibitors and CAR T-cell therapy are changing the treatment landscape and what he expects will be presented at upcoming conferences.

Although the relationship between preceding viral illness and subsequent Guillain-Barre isn’t proven, it is a commonly observed pattern. The severity of GBS ranges from mild sensory disturbances to life-threatening loss of function requiring intubation.

The first treatment for light-chain amyloidosis was approved by the FDA in 2021. Daratumumab-based regimens produce profound hematologic and organ responses, providing a new therapeutic foundation. Early detection, correct fibril typing, the challenges of the very advanced patient, and the lack of therapies to remove amyloid deposits are all being researched but remain elusive.