Ipsen writes down $725M after largest-ever acquisition, trial woes

Less than one year after Ipsen SA made its largest-ever acquisition, the company has written down most of the deal ’s value as a loss.  Ipsen spent $1 billion last year to acquire Canadian startup Clementia Pharmaceuticals Inc. and its leading rare disease drug candidate. But the drug giant, which is based in France and has its U.S. headquarters in Cambridge, recorded a $725 million impairment loss on the de al in its year-end financial report, resulting in a $54 million net loss for Ipsen.  The…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news

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Authors: Laforgia M, Calabrò C, Scattone A, Laface C, Porcelli M, Gadaleta CD, Nardulli P, Ranieri G Abstract Introduction: Mast cell leukemia (MCL) is one of the most aggressive forms of Systemic Mastocytosis (SM), a complex family of rare diseases, for which standard therapies are very few. MCL represents only
Source: Expert Opinion on Pharmacotherapy - Category: Drugs & Pharmacology Tags: Expert Opin Pharmacother Source Type: research
Authors: Quaglino F, Manfrino L, Cestino L, Giusti M, Mazza E, Piovesan A, Palestini N, Lauro C, Castellano E Abstract Parathyroid carcinoma (PC) is a rare disease responsible for about 1% of primary hyperparathyroidism (PHPT) cases. PC usually has an indolent course, tough to differentiate from the benign causes of PHPT, and the only certain diagnosis is histologic. The gold standard surgical treatment is the en bloc resection associated with the homolateral thyroid loboistmectomy. The aim of this study was to underline the main differences between PC and benign PHPT, along with gathering epidemiological knowledge...
Source: International Journal of Endocrinology - Category: Endocrinology Tags: Int J Endocrinol Source Type: research
Abstract Von Hippel-Lindau (VHL) disease is an autosomal dominant inherited tumor syndrome. Clear cell chondrosarcoma (CCCS) is a rare variant of chondrosarcoma. Here, we report a case of CCCS in the talus occurring in a patient with VHL disease. A 32-year-old man presented with ankle pain for 2 years. MRI revealed a 4.2 cm mass in the talus, and needle biopsy was performed. The patient underwent curettage but the final diagnosis was CCCS. Adjuvant radiation therapy was performed without additional talectomy and the patient was alive without recurrence for a follow-up period of 2 years. To our knowledge, this is t...
Source: International Journal of Clinical and Experimental Pathology - Category: Pathology Authors: Tags: Int J Clin Exp Pathol Source Type: research
In this free webinar, Medpace partners with Michelle Eagle of ATOM International, a provider of CE training for clinical trials across the world, to discuss approaches and steps that can be taken to...(PRWeb March 27, 2020)Read the full story at https://www.prweb.com/releases/part_3_rare_disease_clinical_development_strategies_for_ensuring_endpoint_integrity_upcoming_webinar_hosted_by_xtalks/prweb17005830.htm
Source: PRWeb: Medical Pharmaceuticals - Category: Pharmaceuticals Source Type: news
To summarize and extend the phenotypic characterization of Multiple Congenital Anomalies-Hypotonia-Seizures Syndrome, and to discuss genotype-phenotype correlations.
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Gilead asked the U.S. Food and Drug Administration to designate remdesivir a so-called orphan drug, saying it qualified as a rare disease because fewer than 200,000 Americans are infected with the coronavirus.
Source: WebMD Health - Category: Consumer Health News Source Type: news
Authors: Duenas DM, Wilfond BS, Johnson LM PMID: 32208084 [PubMed - in process]
Source: American Journal of Bioethics - Category: Medical Ethics Tags: Am J Bioeth Source Type: research
Artificial intelligence does wonders in healthcare. The technology helped issue the first COVID-19 warning before the WHO and CDC did so. It can slash the phenomenon of alarm fatigue. IBM’s Watson Health leverages the power of A.I. to bring drugs to the market faster. And it does so while cutting costs by over 50%. Speaking of IBM Watson, while the algorithm got its name from the company’s founder Thomas J. Watson, there’s another pop culture figure attached to that name. It’s elementary; we’re talking about none other than Sherlock Holmes’ sidekick, Dr. Watson. It seems like real-wor...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Artificial Intelligence AI digital health Healthcare Medicine technology Source Type: blogs
We report a case of a 50-year-old man with a breast cancer revealed by skin involvement.
Source: Dermatology Online Journal - Category: Dermatology Source Type: research
Aplasia cutis congenita (ACC) is a congenital disorder characterized by localized or generalized absence of skin. Bullous aplasia cutis congenita (BACC) is a rare clinical subtype that has few documented reports in the literature. Herein, we present a new case of BACC in which the bulla was unruptured at birth.
Source: Dermatology Online Journal - Category: Dermatology Source Type: research
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