Saturday Afternoon Live: Dr. Steven Treon’s Presentation at the 2019 IWMF Educational Forum June 8th will be Streamed Online!

• Post author:Trudy Horsting
• Post published:May 23, 2019
• Post category:Rare Disease/Waldenstrom Macroglobulinemia

The International Waldenstrom's Macroglobulinemia Foundation (IWMF) is a nonprofit organization founded by patients. Their mission? Support patients currently living with Waldenstrom's Macroglobulinemia (WM) while simultaneously supporting the search for a…

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Experimental Combination Treatment for Pancreatic Cancer Enters Phase II Trial

• Post author:James Moore
• Post published:May 23, 2019
• Post category:Pancreatic Cancer/Rare Disease

According to a story from Financial Buzz, the drug developer Rafael Pharmaceuticals, Inc., recently announced that a Phase II clinical trial that will test the company's experimental drug CPI-613 in…

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A Look at Progress in Healthcare and a Glimpse into the Future

• Post author:Rose Duesterwald
• Post published:May 23, 2019
• Post category:Chronic Lymphocytic Leukemia/Multiple Myeloma/Myelodysplastic syndromes/Non-Hodgkin's Lymphoma

  The Asco Post recently interviewed Dr. Jean Pierre Bizzari, the Executive Vice President of Celgene, a multinational bioparmaceutical company. The interview covered various aspects of research and development (R&D)…

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Editor’s Choice: Approaching Huntington’s Head On

• Post author:Patient Worthy Contributor
• Post published:May 23, 2019
• Post category:Rare Disease

Happy Thursday! We hope everyone's enjoying the spring weather. Today, we're highlighting a story from a young man advocating for Huntington's patients. Next, we have an article about a surprising…

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Young Girl With Cornelia de Lange Syndrome is Exceeding Expectations

• Post author:James Moore
• Post published:May 23, 2019
• Post category:Cornelia de Lange Syndrome

According to a story from Motherhood In-Style Magazine, Miley Timmermanns was only two years old when she was first diagnosed with an extremely rare disorder: Cornelia de Lange syndrome. Parents…

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Phase 1 Trial of Experimental Treatment for Neuroendocrine Tumors Begins

• Post author:James Moore
• Post published:May 23, 2019
• Post category:Neuroendocrine Tumor/Neuroendocrine Tumors of Gastroenteric or Pancreatic Origin

According to a story from Central Charts, the drug developer Crinetics Pharmaceuticals, Inc. recently issued an announcement revealing that the company has begun the initialization of a Phase 1 clinical…

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Finding Unbiased, Credible Information About Your Rare Disease Can Be Difficult, This Compilation of Resources May Help

• Post author:Trudy Horsting
• Post published:May 23, 2019
• Post category:Ankylosing Spondylitis/Lyme Disease/Systemic Scleroderma/Vasculitis

When you or your child is first diagnosed with a rare disease it can be so difficult to know where to turn. You want to learn everything about the condition,…

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Sanfilippo Syndrome Type B (MPS IIIB) Clinical Trial Continues to Progress

• Post author:James Moore
• Post published:May 23, 2019
• Post category:Sanfilippo Syndrome

According to a story from globenewswire.com, the biopharmaceutical company Abeona Therapeutics, Inc. recently issued an announcement in regards to the company's Phase 1/2 clinical trial, which is testing the company's…

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Positive Results at End of Phase 3 Trial of Candidate Tuberous Sclerosis Complex Drug – CBD

• Post author:Scott Carlson
• Post published:May 23, 2019
• Post category:Tuberous Sclerosis/Tuberous Sclerosis Complex

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…

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How I Turned Huntington’s into My Life’s Passion for Change

• Post author:Patient Worthy Contributor
• Post published:May 23, 2019
• Post category:Huntington's Disease/Rare Disease

My name is Antonio Maltese and I am a 22 year old senior Political Science major concentrating in International Relations and minoring in German at Virginia Commonwealth University. Back in…

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