Rare Diseases definition

Rare Diseases means any disease or condition that meets the criteria for an orphan or rare disease or condition in a Major Market, including, for example in the United States, in §526(a)(2)(1) of the Federal Drug and Cosmetic Act (U.S. Orphan Drug Act (1983)); in Europe, in the Orphan Drug Regulation 141/2000; and in Japan, in the Orphan Drug Amendment (1993).
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Rare Diseases shall include adrenoleukodystophy (ALD) (including but not limited to AMN, CCALD, and all other variants of this disease caused by genetic mutations), beta hemoglobinopathies (including but not limited to beta-thalassemia and sickle cell anemia), [***]. Whether in the case of [***], such diseases shall be considered on a case-by-case basis and considered a Rare Diseases if the incidence or prevalence is similar to those diseases listed as Rare Diseases above. In these situations, Licensee and/or Affiliates shall provide justification as to whether such disease is a Rare Disease, in writing, prior to payment of the Milestone #4 or #5 as applicable.
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Rare Diseases mean human diseases considered to be a “rare disease” in the applicable jurisdiction, e.g., as defined by the Rare Diseases Act of 2002 in the U.S. or by the European Commission on Public Health in Europe. [***] = CERTAIN CONFIDENTIAL INFORMATION CONTAINED IN THIS DOCUMENT, MARKED BY BRACKETS, HAS BEEN OMITTED BECAUSE THE INFORMATION (I) IS NOT MATERIAL AND (II) WOULD BE COMPETITIVELY HARMFUL IF PUBLICLY DISCLOSED.
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Examples of Rare Diseases in a sentence

  • For the breakthrough innovation in complement inhibition, Alexion and Soliris have received the pharmaceutical industry's highest honors: the 2008 Prix Galien USA Award for Best Biotechnology Product with broad implications for future biomedical research and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases.

  • Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI).

  • In November 2019, RDD entered into a non-binding letter of intent to acquire NAIA Rare Diseases (“NAIA”), a privately held biopharmaceutical company developing drugs for Short Bowel Syndrome and other rare gastrointestinal diseases.

  • On November 12, 2019, RDD entered into a nonbinding letter of intent with NAIA Rare Diseases (“NAIA”) to acquire all of the outstanding capital stock of privately-held NAIA in exchange for a combination of cash and shares of the combined company, as well as certain earn-out payments (the “NAIA Acquisition”).

  • Starting from 2003 the Medical Genetics and Rare Orthopedic Diseases Department started a collection of data from patients and families affected by Rare Diseases (in particular skeletal dysplasia) and in the subsequent years stared to standardize this data collection and in collaboration with CLIBI Laboratory established the REM (Registry for Mutliple Ostechondromas Disease).

  • Shire has grown through acquisition, completing a series of major transactions that have brought therapeutic, geographic and pipeline growth and diversification.Shire has four business units that focus exclusively on the commercial execution of its marketed products in the following specialist therapeutic areas: Rare Diseases, Neuroscience, Gastrointestinal and Internal Medicine.

  • We focus on providing treatments in Rare Diseases, Neuroscience, Gastrointestinal, and Internal Medicine and we are developing treatments for symptomatic conditions treated by specialist physicians in other targeted therapeutic areas, such as Ophthalmics.

  • The Pharmaceuticals segment comprises the commercial operations of the following franchises: Specialty Care (Rare Diseases, Multiple Sclerosis, Oncology), Diabetes & Cardiovascular, Established Prescription Products, Consumer Healthcare, and Generics, and dedicated research and development, production and marketing activities for all of Sanofi’s pharmaceuticals operations.

  • Xxxxxxxx Pharma will execute the agreement through its new affiliate Xxxxxxxx Pharma Rare Diseases AG.

  • About Xxxxxxxx Pharma Xxxxxxxx Pharma, owned by Xxxxxxxx Holding, is a pharmaceutical Company committed to helping patients with a constant and prevalent focus on Mental Health, including Pain, Rare Diseases and Consumer Health.

Related to Rare Diseases

  • Occupational disease means a disease contracted in the course of employment, which by its causes and the characteristics of its manifestation or the condition of the employment results in a hazard which distinguishes the employment in character from employment generally, and the employment creates a risk of contracting the disease in greater degree and in a different manner from the public in general.

  • Disease means an alteration in the state of the body or of some of its organs, interrupting or disturbing the performance of the functions, and causing or threatening pain and weakness or physical or mental disorder and certified by a Medical Practitioner.

  • muscular dystrophy means a group of hereditary genetic muscle disease that weakens the muscles that move the human body and persons with multiple dystrophy have incorrect and missing information in their genes, which prevents them from making the proteins they need for healthy muscles. It is characterised by progressive skeletal muscle weakness, defects in muscle proteins, and the death of muscle cells and tissue;

  • sickle cell disease means a hemolytic disorder characterized by chronic anemia, painful events, and various complications due to associated tissue and organ damage; "hemolytic" refers to the destruction of the cell membrane of red blood cells resulting in the release of hemoglobin.

  • Infectious Disease means an illness that is capable of being spread from one individual to another.

  • COVID-19 Pandemic means the SARS-Cov2 or COVID-19 pandemic, including any future resurgence or evolutions or mutations thereof and/or any related or associated disease outbreaks, epidemics and/or pandemics.

  • Infection means the entry and development or multiplication of an infectious agent in the body of humans and animals that may constitute a public health risk;

  • Cannabinoid means any of the chemical compounds that are the active constituents of marijuana.