Category: Rare diseases

Currently, no treatment is available to fix the faulty cilia that cause Primary Ciliary Dyskinesia (PCD), making it a lifelong genetic disorder. However, the crucial question, 'is there medication for PCD?', has a complex answer, as various medicines are used to manage symptoms, prevent infections, and slow disease progression.

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.

According to prescribing information, Onpattro is administered via intravenous infusion once every three weeks by a healthcare professional. This consistent schedule is designed to manage the polyneuropathy symptoms associated with hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Hereditary transthyretin-mediated (hATTR) amyloidosis affects an estimated 50,000 people worldwide [1.5.3, 1.5.4]. This article explores what is the difference between Onpattro and AMVUTTRA, two RNA interference (RNAi) therapies used to treat its symptoms.

The infusion time for Nexviazyme, an enzyme replacement therapy for Pompe disease, can vary significantly among patients, typically lasting several hours depending on dosage and individual tolerance. This intravenous treatment is administered incrementally, with initial infusions often taking longer than subsequent ones. Understanding the factors influencing **how long is a Nexviazyme infusion** is crucial for patients and their caregivers in planning their treatment schedules.

According to the manufacturer, Biocodex, DIACOMIT (stiripentol) is an orphan medicinal product and is distributed exclusively through a specialty pharmacy, confirming it is a specialty drug. This classification significantly impacts how the medication is accessed and managed for patients with Dravet syndrome.

VPRIV (velaglucerase alfa) was approved by the U.S. Food and Drug Administration (FDA) in 2010 for long-term treatment of Type 1 Gaucher disease. For those asking what is the generic for VPRIV, it is important to understand that this medication, as a biologic, does not have a generic version in the traditional sense.

First approved by the FDA in 2002, nitisinone has proven essential for managing rare inherited disorders. For those seeking to know **what is another name for nitisinone**, it is primarily known by its brand names, Orfadin and Nityr, and by the brand Harliku for a specific condition.

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.