According to a press release from Alnylam Pharmaceuticals, the Massachusetts-based biopharmaceutical company has completed the submission of its New Drug Application (NDA) for givosiran — its experimental acute hepatic porphyria…
According to a publication from The Portland Press Herald, Judge Ursula Ungaro, a senior District Judge in the Southern District of Flordia, recently ruled in favor of the United States'…
According to a press release from Danish biopharmaceutical company Orphazyme, the Company intents to proceed with a marketing authorisation application (MAA) for its experimental Niemann-Pick disease type C treatment arimoclomol.…
According to a publication from BNN Bloomberg, regulatory authorities in the European Commission recently granted conditional marketing authorization (CMA) to Zynteglo. Zynteglo is a gene therapy designed to treat beta…
According to a publication from Scleroderma News, a study recently published in Clinical & Transitional Immunology suggests that certain cytokines (small proteins that play an important role in communication between…
According to a publication from Biospace, British clinical drug developer Orchard Therapeutics has secured global intellectual property rights to research, manufacture, and market a gene therapy program for the treatment…
According to a publication from Benzinga, the U.S. Food and Drug Administration has granted Breakthrough Therapy status to Bayer's relapsed follicular lymphoma drug Aliqopa (copanlisib) for the treatment of marginal…
According to a publication from Medical Laboratory Observer, a recently published case study following a family with Diamond-Blackfan anemia presents an overview of the condition; and a multi-step approach describing…
According to a publication on EurekAlert, the National Institutes of Health recently awarded Dr. Jason Karnes, PharmD, PhD (and assistant professor in the University of Arizona College of Pharmacy) with…
In a letter to Dr. Norman Sharpless, the acting commissioner of the Food and Drug Administration since April 5, Senator Ted Cruz encouraged the federal agency to expand the number…
According to a publication from Oncology Nurse Advisor, a study of 109 patients with myeloproliferative neoplasms (certain types of blood cancers) found that mortality rates for younger patients were similar…
For those who haven't finished watching HBO's Game of Thrones, this article contains spoilers for the series finale! If you don't want the ending ruined, read no further! Well, that…
According to a publication from Laboratory Equipment, a new animal study of mice with simulated Huntington's disease suggests that a protein associated with the symptoms of Huntington's can form small,…
A publication at AP Sense outlines some important steps of the early identification and treatment of Addison's disease, a rare condition brought about by the body's underproduction of certain hormones.…
According to a publication from Science Alert, a recent study headed by Dr. Simon Jiang suggests that two rare genetic mutations may be responsible for most cases of lupus. The…
According to a publication from People, Sara Geurts, a 28-year-old model with Ehlers-Danlos syndrome, has been bedridden for several weeks due to complications linked to her condition. Geurts, a Minneapolis…
According to a press release from the British biotechnology company GW Pharmaceuticals, the company has reported successful results at the conclusion of its phase 3 trial of tuberous sclerosis complex…
According to a publication from Express Digest, Britain's National Health Service (NHS) has finally reached an agreement with American biotechnology company Biogen Inc. over the price of Spinraza - a…
According to a publication from Fabry Disease News, the U.S. Food and Drug Administration (FDA) has approved American biotechnology company Avrobio's Investigational New Drug (IND) application for experimental Fabry disease…
This week, a press release from the office of South Carolina senator Lindsey Graham announced the designation of May 17th as Glioblastoma Awareness Day. The Senate Resolution was cosponsored by…
According to a publication from the State University of New York at Buffalo, most symptoms of proximal renal tubular acidosis do not stem from the acidic blood levels that characterize…
According to a publication from ANCA Vasculitis News, a new study in International Kidney Reports suggests that stopping treatment of the disease once it goes into remission may lead to…
According to the Wall Street Journal, the National Institutes of Health (NIH) has reversed its initial decision to prevent two of its researchers from meeting with federal investigators to comment…
Much of what we think counts as "treatment" in medical literature is informed by the amazing successes of high-tech drugs and therapies that have evolved in just the past few…
An Indian health policy initiative meant to provide funding to rare disease patients has struggled to gain approval from the country's administration, according to a publication from DNA India. First…
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