Study Finds That Surgery May be Best for Cushing’s Disease Patients With Unclear MRI Findings
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Study Finds That Surgery May be Best for Cushing’s Disease Patients With Unclear MRI Findings

According to a story from the CushieBlog, a recent study has found that a certain surgical procedure may be the best course of treatment for patients with Cushing's disease whose…

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Congressmen Introduce Bill to Help Chronic Inflammatory Demyelinating Polyneuropathy Patients and Others
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Congressmen Introduce Bill to Help Chronic Inflammatory Demyelinating Polyneuropathy Patients and Others

According to a story from the website of Oregon rep Earl Blumenauer, Blumenauer (D-OR) along with fellow congressmen George Holding (R-NC) and G. K. Butterfield (D-NC) have introduced a bill…

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Program Established by the NIH to Help Rare Disease Advocacy Groups Start Patient Registries

The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…

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Experimental Drug for Sickle Cell Disease Earns Fast Track Designation

According to a story from BioSpace, the biopharmaceutical company Imara, Inc. has recently announced that its experimental product candidate IMR-687 has earned Fast Track designation from the US Food and…

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Transthyretin-Mediated Amyloidosis (ATTR-CM); After Nine Years Pfizer Wins FDA Breakthrough Designation

It has been nine years since Pfizer acquired the drugs vyndagel and vyndamax. According to a report in Biospace, based on the FDA’s recent approval of the two drugs to treat transthyretin-mediated…

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Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation
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Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation

According to a story from BioSpace, the drug developer Cadent Therapeutics recently announced that its investigational product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…

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Boy With Leukodystrophy Gets to Pet His Favorite Animals Thanks to the Make-a-Wish Foundation
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Boy With Leukodystrophy Gets to Pet His Favorite Animals Thanks to the Make-a-Wish Foundation

According to a story from CBS Philly, Jackson Guernsey, who is just two years old, was diagnosed with rare leukodystrophy just six months ago. While the disease has been taking…

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The First Therapy in 15 Years has just Been Approved by the FDA for Malignant Pleural Mesothelioma!

The FDA has just approved the very first therapy for malignant pleural mesothelioma (MPM) in more than 15 years. It is the NovoTTF-100L System, developed by Novocure. Not only is…

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Parents of Kids Affected by Batten Disease Take Legal Action to Maintain Access to Critical Treatment
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Parents of Kids Affected by Batten Disease Take Legal Action to Maintain Access to Critical Treatment

According to a story from Express Digest, Lucy and Mike Carroll don't know for how much longer their children will be able to live happy lives. This is because their…

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Cystic Fibrosis Patient’s Inspiring Speech to New Doctors Stresses The Importance of Patients as Partners

Gunnar Esiason, a 28-year-old cystic fibrosis (CF) patient, just delivered an inspirational pre-commencement speech to the 2019 St. Louis University School of Medicine graduating class. Quite frankly, it contains wisdom…

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CRISPR Uncovers New Gene Fusion as Novel Drug Target for Brain, Ovarian and other Cancers

  CRISPR-Cas9, the genome editing tool, has caused quite a stir in the scientific community. According to a recent article in Science Daily, with results published in Nature Communications, CRISPR has…

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Mouse Model Continues to Show Efficacy of GKT831 for Treating Cholestatic Fibrosis in Primary Biliary Cholangitis

Preclinical data supporting the efficacy of GKT831 as a potential therapy for cholestatic fibrosis has just been published in the Journal of Hepatology. GKT831 GKT831 is both a NOX1 enzyme and…

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