Patient Advocacy Groups Call for Indian Health Ministry to Implement Interim Care Policy for Rare Disease Patients
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Patient Advocacy Groups Call for Indian Health Ministry to Implement Interim Care Policy for Rare Disease Patients

According to a recent publication from Moneycontrol, patient advocacy groups in India are calling for the country's Minister of Health and Family Welfare, Harsh Vardhan, to establish a provisional system…

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Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome
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Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome

According to a publication from the Korea Times, the Eone Diagnomics Genome Center in Incheon, South Korea, is working to improve patients' accessibility to their own genetic sequence. Researchers believe…

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A Recent Discovery Could Make Cancer Treatments More Effective
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A Recent Discovery Could Make Cancer Treatments More Effective

According to a story from news-medical.net, scientists may have made a significant breakthrough discovery that could make cancer treatment more effective. The discovery involves a new method that breaks through…

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FDA Begins Priority Review of Xtandi, a Drug Candidate for Metastatic Hormone-Sensitive Prostate Cancer

According to a press release from American pharmaceutical giant Pfizer, Inc. the FDA recently agreed to review a supplemental New Drug Application for Xtandi. Xtandi was first approved for the…

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FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy
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FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy

According to a press release from American biotechnology company Fortress Biotech, the American Food and Drug Administration (FDA) recently awarded Regenerative Medicine Advanced Therapy designation to the experimental gene therapy…

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Beta Blocker Fails to Stop Hereditary Hemorrhagic Telangiectasia-Associated Nosebleeds
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Beta Blocker Fails to Stop Hereditary Hemorrhagic Telangiectasia-Associated Nosebleeds

According to a recent study published in Nature Research, a team of scientists studied the efficacy of timolol in treating nosebleeds associated with hereditary hemorrhagic telangiectasia. Timolol is a beta…

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ICYMI: An Experimental Treatment for POMC and LEPR Deficiency Obesity Appears Effective

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., has recently announced the results from two phase 3 clinical trials. These trials were testing the company's experimental…

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CPRIT Awards Emtora Biosciences $3 Million Grant to Develop Investigational Familial Adenomatous Polyposis Drug
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CPRIT Awards Emtora Biosciences $3 Million Grant to Develop Investigational Familial Adenomatous Polyposis Drug

According to a recent press release, Texas-based biotechnology company Emtora Biosciences was chosen earlier this month to receive a $3 million grant from the Cancer Prevention and Research Institute of…

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Good Morning America Spotlights Girl with Rare Disease Mesmerized by Inclusive Beauty Ad

Recently, Good Morning America demonstrated the importance of showing children with disabilties examples of diversity in the media. Carolyn Anderson shared with the popular morning show how her 4-year-old-daughter Maren…

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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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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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