Tag: Rare disease

Overactive complement system activity is a primary driver of several rare autoimmune diseases. For individuals with these challenging conditions, understanding *why do you take Ultomiris* is crucial to managing symptoms, preventing organ damage, and improving quality of life. This long-acting medication specifically targets a key protein in this immune pathway to provide sustained disease control.

As of a recent pipeline update, Pfizer had over 100 drug candidates in active development. This extensive portfolio highlights **what drugs does Pfizer have in the pipeline**, showcasing a strategic focus on next-generation medicines and technologies across multiple therapeutic areas.

In May 2019, the FDA approved Tafamidis, marking the first time a medication was specifically approved to treat transthyretin amyloid cardiomyopathy (ATTR-CM). This article explores the specific clinical scenarios for which this groundbreaking drug is prescribed, detailing the disease it targets and its significant impact on patient outcomes.

With more than 7,000 rare diseases known worldwide, affecting an estimated 300 million people, Pfizer's focus on what is Pfizer rare disease represents a significant commitment to an area of high unmet medical need. For over thirty years, the company has worked to provide critical treatment options for patients with rare conditions, advancing research across areas like rare hematology, neurology, and cardiology.

With a list price of over $2 million per dose, Zolgensma is one of the most expensive medications in the world. This staggering cost raises a critical question for families affected by spinal muscular atrophy (SMA): who pays for Zolgensma? The answer involves a complex combination of health insurance coverage, manufacturer assistance programs, and innovative payment models.

Hereditary angioedema (HAE) medication costs can exceed hundreds of thousands of dollars per patient annually, with some treatments carrying a list price of over $600,000 per year. The question, **how much does hereditary angioedema medication cost?**, is complex, as the financial burden is influenced by the type of treatment, insurance coverage, and available patient assistance programs.

Affecting fewer than 200,000 people in the United States, the porphyrias are a group of rare disorders [1.7.2]. A key question for understanding this group of conditions is, **what is aminolevulinic acid disease?** This term refers to these disorders where aminolevulinic acid (ALA) accumulates.

The U.S. Food and Drug Administration (FDA) approved Kebilidi in November 2024, approximately two years after the same gene therapy was approved in the European Union (EU) as Upstaza. This means that when asking, 'Is kebilidi the same as upstaza?', the answer is yes: they are different brand names for the exact same medical product, `eladocagene exuparvovec`.

Synthesized in 1916 for the treatment of parasitic diseases, suramin is not commercially available to the general public in the United States. The answer to 'Is suramin available in the US?' is complex and involves highly controlled distribution channels for specific medical needs.

The medication canakinumab, marketed under the brand name Ilaris®, was first approved by the U.S. Food and Drug Administration (FDA) in June 2009. So, who makes canakinumab? The Swiss multinational healthcare company Novartis is the developer and manufacturer of this important biopharmaceutical.