Ontario Focused on Rare Disease Drugs Ahead of National Drug Plan Talks Ontario Focused on Rare Disease Drugs Ahead of National Drug Plan Talks

The Canadian province of Ontario will push for the federal government to tackle the high cost of treatments for rare diseases as negotiations over a new national prescription drug program are set to kick off, the province's health minister told Reuters on Monday.Reuters Health Information
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Family Medicine/Primary Care News Source Type: news

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ConclusionGiant hydronephrosis can present rare symptoms as “intractable hiccups”. The selection of treatment should be made depending on the cause.
Source: International Journal of Surgery Case Reports - Category: Surgery Source Type: research
ConclusionThe LMS should be taken under consideration when diagnosing a tumor of the GB.
Source: International Journal of Surgery Case Reports - Category: Surgery Source Type: research
French drugmaker has diagnosed oncology and rare diseases as potential areas of growth
Source: FT.com - Drugs and Healthcare - Category: Pharmaceuticals Source Type: news
Publication date: Available online 12 December 2019Source: Autoimmunity ReviewsAuthor(s): Ruth Percik, Gadi Shlomai, Amir Tirosh, Amit Tirosh, Raya Leibowitz-Amit, Yael Eshet, Gahl Greenberg, Alex Merlinsky, Ehud Barhod, Yael Steinberg-Silman, Tal SellaAbstractObjectiveImmune checkpoint inhibitors have introduced a new and heterogeneous class of immune-related adverse effects, with the endocrine system being a predominant target for autoimmunity. Autoimmune hypothalamic-pituitary-adrenal axis (HPA) diseases induced by checkpoint inhibitors are being increasingly recognized. We aimed to characterize the spectrum of checkpoi...
Source: Autoimmunity Reviews - Category: Allergy & Immunology Source Type: research
Pfizer Inc said on Friday the European Medicines Agency granted the company a positive recommendation for approval of its treatment for a rare disorder that occurs in patients with cardiomyopathy.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Conclusion: Although left-sided gallbladder is a very rare disease, it is possible to diagnose it preoperatively and perform laparoscopic cholecystectomy safely by adjusting port position. The common important features of left-sided gallbladder include distribution of the left portal vein crossing over to the right side of the liver and increased size of the left portal vein. These variations may have important clinical implications in the management of hepatic resection including donor hepatectomy. PMID: 31824885 [PubMed]
Source: Annals of Surgical Treatment and Research - Category: Surgery Tags: Ann Surg Treat Res Source Type: research
The FDA unexpectedly reversed course Thursday, approving the second treatment developed by Sarepta Therapeutics for the rare disease Duchenne muscular dystrophy just a few months after rejecting the drug.   Sarepta (Nasdaq: SRPT) announced Thursday evening that the agency had approved its second drug, called Vyondys 53. After the drug was originally rejected by the FDA in August due to concerns over the risk of infection at the infusion site and renal toxicity, the company filed an appeal. The comp any…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
Abstract OBJECTIVE: Klippel-Feil syndrome (KFS) is characterized by congenital fusion of the cervical vertebrae. Due to its rarity, minimal research has been done to assess the quality and management of pain associated with this disorder. Using a large global database, the authors report a detailed analysis of the type, location, and treatment of pain in patients with KFS. METHODS: Data were obtained from the Coordination of Rare Diseases at Stanford registry and Klippel-Feil Syndrome Freedom registry. The cervical fusions were categorized into Samartzis type I, II, or III. The independent-sample t-test, Wilc...
Source: Journal of Neurosurgery.Spine - Category: Neurosurgery Authors: Tags: J Neurosurg Spine Source Type: research
CAMBRIDGE, Mass., Dec. 12, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Vyondys 53™...
Source: Drugs.com - New Drug Approvals - Category: Drugs & Pharmacology Source Type: news
Some people with rare diseases rely on peer-led support groups for disease-specific education and emotional and practical support. Systemic sclerosis (SSc), or scleroderma, is a rare autoimmune connective tiss...
Source: Trials - Category: General Medicine Authors: Tags: Study protocol Source Type: research
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