PointR Data Merges into Mateon Therapeutics — Company Will Focus on Using Data Analysis and AI to Develop New Treatments
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PointR Data Merges into Mateon Therapeutics — Company Will Focus on Using Data Analysis and AI to Develop New Treatments

According to a press release from Mateon Therapeutics, Mateon's recent merger agreement with PointR Data establishes plans to combine the use of artificial intelligence and data collection for the development…

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Amicus Therapeutics Continue to Develop Investigative Treatments for Fabry, Pompe, and Batten Diseases
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Amicus Therapeutics Continue to Develop Investigative Treatments for Fabry, Pompe, and Batten Diseases

According to a publication from Fabry Disease News, Amicus Therapeutics has as many as 14 investigational drugs currently in development for the treatment of rare diseases including Fabry, Pompe, and…

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First Human Trial Completed for Experimental Scleroderma and Multiple Sclerosis Drug
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First Human Trial Completed for Experimental Scleroderma and Multiple Sclerosis Drug

According to a story from Financial Buzz, the drug developer Emerald Health Pharmaceuticals, Inc., recently announced that the company had completed a phase 1 clinical trial for its experimental drug…

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New Study Suggests as Many as Half of Systemic Sclerosis Patients Affected by Sarcopenia
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New Study Suggests as Many as Half of Systemic Sclerosis Patients Affected by Sarcopenia

According to a publication from Scleroderma News, a recent study published in Rheumatology International suggests that the majority of systemic sclerosis patients experience sarcopenia as a result of their condition. Perhaps…

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CTD Holdings Gives Presentation on Investigatory Niemann-Pick Type C Drug at Family Support and Medical Conference

According to a press release from CTD Holdings, a biotechnology company based in Gainesville, Florida, the Company recently presented data collected from their investigatory Niemann-Pick Disease treatment, Trappsol Cyclo, to…

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Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation
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Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company CrystalGenomics, Inc. recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational drug candidate CG-745 Orphan…

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Clinical Trial Shows POEM Procedure is Less Invasive and More Effective for Achalasia Treatment

Achalasia Achalasia is a rare disease which causes damage to the nerves in the esophagus. Patients have difficulty swallowing, heartburn, and chest pain. Many also experience weight loss. While there is…

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The Results of This Phase 3 PKAN Clinical Trial are a Major Letdown
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The Results of This Phase 3 PKAN Clinical Trial are a Major Letdown

According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently released the topline results from its phase 3 clinical trial. This clinical trial was testing the company's drug…

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The Isaac Foundation has Turned to Television to Get Their Mucopolysaccharidosis Message Heard

The Isaac Foundation The Isaac Foundation was established by a man named Andrew McFadyen. He created the organization in 2007 in honor of  his son who is diagnosed with the rare…

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Expectations For This Clinical Trial Were to Slow Down ALS Progression; Instead it Reversed One Man’s Symptoms

  According to a recent article published in News NWA, in 2017 Mark Bedwell, of Fort Smith, Arkansas had been told that based upon the rapid progression of his amyotrophic…

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The Drug Fasenra has Earned Orphan Drug Designation to Treat Eosinophilic Esophagitis
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The Drug Fasenra has Earned Orphan Drug Designation to Treat Eosinophilic Esophagitis

According to a story from Check Orphan, the biopharmaceutical company AstraZeneca recently announced that its drug benralizumab (marketed as Fasenra) has earned Orphan Drug designation from the US Food and…

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Medical Student Keeps Pursuing Her Dream with CIDP and CRPS
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Medical Student Keeps Pursuing Her Dream with CIDP and CRPS

According to a story from National Pain Report, Jill Konowich was well on her way to becoming a medical doctor. She was studying at the Rutgers New Jersey Medical School.…

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After Being Part of a Crucial Spinal Muscular Atrophy Trial, a Family’s Future Remains Uncertain

According to a story from Palo Alto Online, it was only a few months into the life of Aleksandr Kostanyan when parents Svetlana Sargsyan and Tigran Kostanyan realized that something…

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Abivax Begins Enrollment in Phase 2b Study of Experimental Ulcerative Colitis Treatment
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Abivax Begins Enrollment in Phase 2b Study of Experimental Ulcerative Colitis Treatment

According to a press release from French biotechnology company Abivax, the Company has successfully begun recruitment for its phase 2b clinical study of ABX464, its experimental once-a-day ulcerative colitis medication.…

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ICYMI: With the FDA’s Third Approval for Tissue Agnostic Therapies, Oncologists are Headed to the Next Level in Cancer Treatment

Since the 1940s oncologists have been focusing on tumors at the location in the body where the tumors originated. According to a recent FDA press release, this is the third…

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IND Accepted for an Experimental Sanfilippo Syndrome Treatment
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IND Accepted for an Experimental Sanfilippo Syndrome Treatment

According to a story from forextv.com, the biopharmaceutical company Seelos Therapeutics, Inc. has recently announced that the US Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND)…

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Experimental Treatment for Immune Thrombocytopenia Begins Trials in China
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Experimental Treatment for Immune Thrombocytopenia Begins Trials in China

According to a story from uk.finance.yahoo.com, the company Hutchison China MediTech Limited ("Chi-Med") has recently kicked off a phase 1 clinical trial. This trial is testing the company's investigational drug…

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Rapid Whole Genome Sequencing is Getting Patients With Rare Genetic Disorders Diagnosed More Quickly
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Rapid Whole Genome Sequencing is Getting Patients With Rare Genetic Disorders Diagnosed More Quickly

According to a story from the National Center for Advancing Translational Sciences (NCATS), research supported by the center is making it possible for kids born with serious, rare genetic disorders…

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