Orphan Disease definition

Orphan Disease means (a) PD; (b) any disease or condition with a patient prevalence of (i) less than 200,000 in the United States, or (ii) not more than 5 in 10,000 in Europe, or (iii) less than 50,000 in Japan, in each case, whether or not a product intended to treat such disease or condition has achieved an orphan drug designation; or (c) any disease or condition for which a product intended to treat such disease or condition has received orphan drug designation from any Regulatory Authority in the United States, Europe or Japan. For clarity, Orphan Disease shall not include Alzheimer’s disease.
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Orphan Disease means a disease or condition for which in any given country a pharmaceutical treatment meets, as of the Effective Date, the definition of an “orphan product” or “orphan drug” for treatment of a “rare disease” under the U.S. Orphan Drug Act or Regulation No. 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products, each as amended, or any successor laws or regulations thereto.
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Orphan Disease means (a) PD; (b) any disease or condition with a patient prevalence of (i) less than 200,000 in the United States, or
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Examples of Orphan Disease in a sentence

  • Our efforts in Xxxxxxxxx’x disease are being extended into other Xxxxxxxxx’x-related indications, such as the Orphan Disease Multiple Systems Atrophy, or MSA.

  • Potential subjects will be identified through existing genetic research studies at Children’s Hospital Boston, such as Xx. Xxxxx' studies on the molecular basis of neuromuscular disease and the Manton Center for Orphan Disease Research, or directly through one of the investigators.


More Definitions of Orphan Disease

Orphan Disease means a disease or condition for which in any given country a pharmaceutical treatment meets, as of the Effective
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Orphan Disease means a condition that affects fewer than 200,000 people nationwide. This includes diseases such as cystic fibrosis, Xxx Xxxxxx’x disease, Tourette’s syndrome, Hamburger disease, Job syndrome, and acromegaly, or “gigantism.”
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Related to Orphan Disease

  • orphan means a child who has no surviving parent caring for him or her;

  • 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;

  • 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.

  • Diagnosis means the definition of the nature of the Client's disorder. When formulating the Diagnosis of Client, CONTRACTOR shall use the diagnostic codes and axes as specified in the most current edition of the DSM published by the American Psychiatric Association. DSM diagnoses will be recorded on all IRIS documents, as appropriate.

  • inherited metabolic disease means a disease caused by an inherited abnormality of body chemistry for which testing is mandated by law;

  • 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.

  • Medical marijuana product means a product that contains cannabinoids that have been extracted from plant material or the resin therefrom by physical or chemical means and is intended for administration to a licensed patient, including but not limited to concentrates, oils, tinctures, edibles, pills, topical forms, gels, creams, and other derivative forms, except that this term does not include live plant forms.

  • Collaboration Compound means any of the following: (a) FG-4592, (b) any HIF Compound (other than FG-4592) that is added to this Agreement pursuant to Section 3.6, and (c) any salts, esters, complexes, chelates, crystalline and amorphous morphic forms, pegylated forms, enantiomers (excluding regioisomers), prodrugs, solvates, metabolites and catabolites of any of the foregoing ((a) or (b)).

  • Opioid antidote means any drug, regardless of dosage amount or method of administration, which has been approved by the United States Food and Drug Administration (FDA) for the treatment of an opioid overdose. “Opioid antidote” includes, but is not limited to, naloxone hydrochloride, in any dosage amount, which is administered through nasal spray or any other FDA-approved means or methods.

  • Terminal disease means an incurable and irreversible disease that has been medically confirmed and will, within reasonable medical judgment, produce death within six months.

  • Diagnostic Product means In Vitro Diagnostics, In Vivo Diagnostic Agents and any product used for Diagnosis.

  • Collaborative drug therapy management means participation by an authorized pharmacist and a physician in the management of drug therapy pursuant to a written community practice protocol or a written hospital practice protocol.

  • Biological diversity means the variability among living organisms from all sources including, inter alia, terrestrial, marine and other aquatic ecosystems and the ecological complexes of which they are part; this includes diversity within species, between species and of ecosystems.

  • Medical cannabis means the same as that term is defined in Section 26-61a-102.

  • Behavioral therapy means interactive therapies derived from evidence-based research, including applied behavior analysis, which includes discrete trial training, pivotal response training, intensive intervention programs, and early intensive behavioral intervention.

  • HIV means human immunodeficiency virus.

  • Collaboration Product means a pharmaceutical product containing or comprising Compound in any dosage form alone, or in combination with, one or more other pharmaceutically active ingredients, and any and all Improvements thereto.

  • Licensed Compounds means any EZH2 Compound(s) that is:

  • Gene Therapy means the introduction of a nucleic acid sequence encoding a protein intended for or otherwise conferring therapeutic benefit into a person for therapeutic purposes (i) by in vivo introduction for incorporation into cells of such person, or (ii) by ex vivo introduction into cells for transfer into a person.

  • Commercialization or “Commercialize” means activities directed to marketing, promoting, research and development as required, manufacturing for sale, offering for sale, distributing, importing or selling a product, including sub-licensing or sub-contracting of these activities.

  • Product Candidate means each of MYO-101, MYO-102, MYO-103, MYO-201 and MYO-301, as defined on Exhibit B, in each case in any form or formulation.

  • Behavioral health disorder means either a mental disorder

  • Collaboration Patents means any and all Patents that claim or cover any of the Collaboration Know-How.

  • Collaboration Technology means all Collaboration Patents and Collaboration Know-How.

  • Diagnosis of autism spectrum disorder means medically necessary assessments, evaluations, or tests

  • Therapeutic school means a residential group living facility: