Tag: Rare disease treatment

In September 2023, the FDA approved Rivfloza® (nedosiran), marking a significant milestone for patients with primary hyperoxaluria type 1 (PH1), a rare genetic disorder characterized by excessive oxalate production. As an innovative RNA interference (RNAi) therapy, Rivfloza works to reduce the burden of oxalate by targeting the source of its overproduction in the liver.

Annual costs for alpha-1 antitrypsin (AAT) augmentation therapy can be substantial, with some insurer costs exceeding $80,000 to $100,000 per year. Understanding **what is the price of alpha 1 antitrypsin injection?** is a complex matter, as the final cost depends on various medical and financial variables for each patient.

With a price tag of $4.25 million for a single treatment, Lenmeldy, a gene therapy for metachromatic leukodystrophy (MLD), is the highest-priced drug in the U.S.. This staggering figure brings critical attention to the complex market of specialized medicines, forcing us to ask: which drug has the highest price and why?.

Approved by the FDA in November 2020, Oxlumo was the first-ever therapy for Primary Hyperoxaluria type 1 (PH1). For those wondering, what is the generic name for Oxlumo?, the answer is lumasiran, a targeted RNA interference (RNAi) therapeutic designed to treat this rare genetic disorder.

In September 2024, the U.S. Food and Drug Administration (FDA) approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC), classifying it as a modified amino acid. This orally administered medication addresses the progressive neurological decline seen in this rare genetic disorder.

The annual list price for ONPATTRO (patisiran) is approximately $450,000, but the net price after discounts can be closer to $345,000 [1.2.2, 1.2.3]. This article explores the details of **how much does ONPATTRO cost per year**, including factors that influence its price and available financial support.

According to reputable drug information databases like Drugs.com, the term 'Fab Tablet' is not a standard medication name and can lead to significant confusion, as 'Fab' is a component of several different product names with widely varying uses. This article clarifies what is the use of Fab Tablet by distinguishing between common supplements and specialized prescription drugs that feature 'Fab' in their branding.

The list price for an average patient on Koselugo is estimated to be approximately $12,500 per month, though this figure can change based on dosage and is often reduced significantly through insurance and patient assistance. This high price tag makes understanding how much does Koselugo cost a critical step for families managing neurofibromatosis type 1.

Over 6,000 patients worldwide are part of the International Collaborative Gaucher Group Gaucher Registry, which has provided long-term data supporting the use of Cerezyme (imiglucerase). This therapy is specifically approved for treating certain types of Gaucher disease and targets the debilitating symptoms associated with the condition. Understanding **what are the indications for Cerezyme** is crucial for patients and healthcare providers managing this rare genetic disorder.

Affecting approximately 3,500 people in the US, Pompe disease is a rare genetic disorder treated by enzyme replacement therapies (ERTs). **What is Nexviazyme** and how does this second-generation treatment, developed by Sanofi, offer a new option for patients with the late-onset form of the disease?.