Rare Disease Research, Treatment, Advances - Pfizer
With more than three decades of experience in rare diseases, our broad portfolio aims to address unmet medical needs across four main therapeutic areas: rare hematology, rare endocrine/metabolic, rare cardiology, and rare neurology.
Rare Disease Awareness, Support & Advocacy | Pfizer
For over 30 years, Pfizer Rare Disease has worked hand in hand with the rare disease community to forge a pathway toward life-changing innovations. We’ve supported numerous patients and their families on their journey toward healthier futures.
Rare Disease Drug Pipeline and Clinical Trials | Pfizer
Feb 10, 2020 · Pfizer Rare Disease is focused on developing recombinant adeno-associated virus (rAAV) precision-targeting gene therapies, because of its potential to consistently target cells with treatment.
Alexion, AstraZeneca Rare Disease, enters agreement with Pfizer …
Jul 28, 2023 · Alexion, AstraZeneca Rare Disease, today announced that it has entered a definitive purchase and licence agreement for a portfolio of preclinical gene therapy programmes and enabling technologies from Pfizer Inc. (Pfizer).
On Rare Diseases Day, New Partnerships and Investments Could …
Mar 3, 2022 · Pfizer’s rare disease pipeline has more than 10 investigational medicines in clinical development, with more than five new molecular entities and three gene therapy programs in Phase 3.
Pfizer Rare Disease is currently focusing on diseases that have single gene alterations, such as hemophilia A, hemophilia B, and Duchenne muscular dystrophy (DMD).
Rare diseases cover a wide variety of conditions: neurological disorders, infectious diseases, rare cancers, hematological conditions, endocrine disorders, autoimmune disorders, respiratory diseases, and a variety of genetic disorders.
FDA approves Pfizer’s first gene therapy for rare inherited …
Apr 26, 2024 · The Food and Drug Administration on Friday announced that the agency approved Pfizer 's treatment for a rare genetic bleeding disorder, making it the company's first gene …
Rare Disease - Pfizer UK
There are an estimated 10,000 rare diseases in the world today and counting. While these diseases may be individually rare they are collectively common, affecting approximately 3.5 million people in the UK alone.
Pfizer explores options for some rare disease, cancer drugs
Jan 6, 2023 · Pfizer Inc is exploring options for some early-stage treatments for rare diseases and cancer in a bid to focus on "high-impact" medicines and vaccines, the company said on Thursday.