Tag: Rare disease

Idebenone is a synthetic analog of coenzyme Q10, primarily used to address cellular energy deficiencies and oxidative stress in certain genetic disorders. Therefore, understanding **what is Idebenone 45 mg used for** involves recognizing that this dosage unit is part of a regimen tailored to specific conditions.

Remodulin is a specialized medication primarily used to treat pulmonary arterial hypertension (PAH), a rare but serious condition. As a **prostacyclin analog**, it mimics a naturally occurring substance in the body to widen blood vessels in the lungs and reduce the workload on the heart. This powerful therapy is often reserved for patients with more severe symptoms.

A 28-day course of Vijoice (alpelisib) had a wholesale cost of $32,500 in 2022, highlighting the significant financial burden associated with this targeted therapy. This article explores how much alpelisib costs in the US under its different brand names, the factors affecting price, and available financial support.

Affecting approximately 300 million people worldwide, rare diseases present an immense unmet medical need that pharmaceutical companies like Johnson & Johnson (J&J) are working to address. At the heart of J&J's strategy for autoantibody-mediated rare diseases is its FcRn inhibitor, nipocalimab, now known by the brand name Imaavy, which received FDA approval for myasthenia gravis in 2025.

In a pivotal Phase III clinical trial, 86% of patients treated with Isturisa maintained normal cortisol levels at week 34 during the randomized withdrawal period. So, what is the drug Isturisa used for? It is an oral medication prescribed to treat endogenous Cushing's syndrome in adults.

In clinical trials, the Orladeyo treatment demonstrated a significant reduction in the rate of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 and older. As the first oral medication for HAE prophylaxis, it offers a convenient and effective option for managing this rare genetic disorder.

Takeda Pharmaceuticals is the current manufacturer of Cinryze, but the medication has a rich history involving several pharmaceutical companies. First developed by ViroPharma, the rights to Cinryze were later acquired by Shire before eventually being integrated into Takeda's portfolio following a major corporate acquisition. This complex journey reflects the consolidation common within the modern pharmaceutical industry.

Approved by the FDA in 2007, Kuvan (sapropterin dihydrochloride) is a prescription medication used to address the genetic disorder phenylketonuria (PKU). This treatment specifically targets patients with BH4-responsive PKU, lowering their blood phenylalanine levels in conjunction with a restricted diet.

With a price tag of $4.25 million per one-time treatment, Lenmeldy, a gene therapy for a rare genetic disorder, holds the record for **what is the most expensive drug ever sold**. This staggering cost for a single dose highlights the unprecedented pricing of modern gene therapies and raises important questions about healthcare affordability and access.

Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) is a rare and progressive genetic disorder, estimated to affect between 10,000 and 40,000 people globally. This condition is caused by a gene mutation that results in abnormal protein deposits that damage the nervous system and other vital organs.