Medical products for rare diseases and conditions
Orphan medicinal products - Public Health - European Commission
Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in ...
In the United States, over 400 products have been approved as therapy for more than 200 rare disease indications and, in the European Union, over 70 products ...
Rare Diseases - Cerba Research
In the past 5 years, we've conducted 190+ rare disease trials, resulting in 36 drug approvals for rare to ultra-rare conditions. Get in touch to learn more.
Focusing on these uncommon and underserved medical conditions, Sanofi's Rare Diseases franchise is committed to offering sustainable, potentially ...
The first therapies available to treat many rare pediatric diseases, including a progressive, metabolic disease called hypophosphatasia (HPP), an inherited ...
Chiesi Global Rare Diseases | A Rare Disease Therapeutics Company
Chiesi Global Rare Diseases is a family-owned and run biopharmaceutical company with a commitment to providing transformative care to patients with rare ...
Working Group on MedTech for Rare Diseases - IRDiRC
The Working Group will primarily focus on devices used for either the treatment of rare diseases, such as implants, or devices used to support physical ...
Small Data Challenges of Studying Rare Diseases - JAMA Network
... pharmaceutical and medical device companies to develop new treatments for patients with rare diseases. ... products-rare-diseases-conditions. 4.
Criteria to define rare diseases and orphan drugs - BMJ Open
Introduction Rare diseases (RDs) are often chronic and progressive life-threatening medical conditions that affect a low percentage of the population compared ...
Inflation Reduction Act is Impacting Rare Disease Patients
Orphan drugs are products used to treat, prevent, or diagnose a disease that affects fewer than 200,000 people in the United States or that will ...
Treating rare diseases: How digital technologies can drive innovation
Pharmaceutical companies are using recent advances in digital technologies to improve care strategies and provide hope for rare disease ...
Orphan Drug - an overview | ScienceDirect Topics
Orphan medicines are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases. They are 'orphans' ...
Rare Diseases - Takeda Pharmaceuticals
Takeda aspires to transform the treatment of rare diseases in immunology, hematology, metabolic and lysosomal storage disorders. These rare genetic and ...
Orphan Devices for the Surgical Treatment of Patients with Rare ...
Orphan devices are medical devices intended for the treatment of a very rare life-threatening or chronically debilitating condition.
Orphan Drug Label Expansions: Analysis Of Subsequent Rare And ...
We found that 491 novel orphan drugs were approved between 1990 and 2022. To date, 65 percent have been approved for a single rare disease, 15 ...
Orphan Drugs | Dawnbreaker MRR
Since 1983, the U.S. Food & Drug Administration (FDA) has approved more than 650 therapies for rare diseases and conditions. Since 1990, the FDA has approved 72 ...
Drug development for ultra-rare diseases: What happens when N=1?
By treating ASOs for new ultra-rare diseases as repurposed drugs, the FDA can reduce the amount of toxicology and preclinical studies needed to ...
FDA Designations For Rare Disease Products, Part 2: Orphan Drug ...
FDA Designations for Rare Disease Products, Part 2: Orphan Drug Designation · The product must be intended for use in a rare disease or ...
By changing medical practice, pursuing first or best-in-class medicines, focusing on cutting edge research and development and utilising new digital health ...
Global health for rare diseases through primary care - The Lancet
Recognising this opportunity, the International Rare Diseases Research Consortium (IRDiRC) assembled a global, multistakeholder task force to ...