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We believe that patients should have access to innovative medicines.

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About PhRMA

The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading innovative biopharmaceutical research companies, which are laser focused on developing innovative medicines that transform lives and create a healthier world. Together, we are fighting for solutions to ensure patients can access and afford medicines that prevent, treat and cure disease. Over the last decade, PhRMA member companies have invested more than $850 billion in the search for new treatments and cures, and they support nearly five million jobs in the United States.

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For information on how innovative medicines save lives, please visit:  
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Blood disorders often require lifelong treatment involving routine drug infusions or blood transfusions. They can also be associated with frequent visits to physician offices, infusion centers or even the emergency room. Patients with these disorders often experience debilitating pain, disability, reduced life expectancy and quality of life. Potential gene therapies may offer long-term benefits and even cures for patients with blood disorders with a single administration. Since they may drastically reduce the burden of the current standard of care, they may also:

  • Reduce the need for patients to miss work for treatment and/or symptoms, leading to economic benefits for patients
  • Dramatically reduce existing treatment burdens for patients
  • Result in savings for the health care system

Gene Therapies Can Bring Hope to Patients with Blood Disorders image

download Beta Thalassemia One Pager

download gene therapy one pager

download Hemophilia A One Pager

download Hemophilia B One Pager

download Sickle Cell One Pager

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Report

Disease

Gene Therapies Can Bring Hope to Patients with Blood Disorders

May 18, 2022

Gene Therapies Can Bring Hope to Patients with Blood Disorders image

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