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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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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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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Ulcerative Colitis Patients are at Higher Risk for Liver Disease, Study Finds

• Post author:James Moore
• Post published:May 22, 2019
• Post category:Rare Disease/ulcerative Colitis

According to a story from MD Magazine, a recent study that was presented at Digestive Disease Week 2019 suggests that patients with ulcerative colitis, a rare form of inflammatory bowel…

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Don’t Miss this Q&A about the Upcoming NORD Patient and Family Forum

• Post author:Patient Worthy Contributor
• Post published:May 22, 2019
• Post category:Rare Disease

Q&A with NORD Director of Membership, Debbie Drell, on the Living Rare, Living Stronger Patient and Family Forum, June 21-23 in Houston, Texas Which health care professionals would most benefit…

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Outbreaks of Infectious Disease Often Leave The Chronically Ill Most Vulnerable

• Post author:James Moore
• Post published:May 21, 2019
• Post category:Rare Disease

According to a story from The Washington Post, a host of different factors ultimately led to the tragic death of Olivia Shea Paregol, a freshman at the University of Maryland.…

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England’s NHS Reaches Agreement for Coverage with SMA Drug Manufacturer Biogen

• Post author:Scott Carlson
• Post published:May 21, 2019
• Post category:Rare Disease/Spinal Muscular Atrophy

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…

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Experimental Treatment for Multiple Myeloma Earns Orphan Drug Designation

• Post author:James Moore
• Post published:May 21, 2019
• Post category:Multiple Myeloma/Rare Disease

According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…

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This Spinocerebellar Ataxia Patient is Committed to Spreading Awareness

• Post author:James Moore
• Post published:May 21, 2019
• Post category:Rare Disease/Spinocerebellar Ataxia

According to a story from Kamloops Coffee Talk, Darrin Rein, of Kamloops, British Columbia, was just recently diagnosed with spinocerebellar ataxia, a rare disease that affects mobility and coordination. Despite…

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The FDA Has Just Approved Teduglutide for Children with Short Bowel Syndrome

• Post author:Trudy Horsting
• Post published:May 21, 2019
• Post category:Rare Disease/Short bowel syndrome

Short Bowel Syndrome Short bowel syndrome (SBS) is a rare condition which occurs when patients have a large portion of their intestine removed. The surgery which leads to this diagnosis…

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Could Stopping Using the Word “Rare” Increase the Rate of Diagnosis for Rare Disease Patients?

• Post author:Trudy Horsting
• Post published:May 21, 2019
• Post category:Alport syndrome/Duchenne Muscular Dystrophy/Friedreich's Ataxia/Rare Disease

The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…

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Resort City in California has Made Their Beaches Wheelchair Accessible with Temperature Controlled Mats

• Post author:Trudy Horsting
• Post published:May 20, 2019
• Post category:Rare Disease

Coronado is a resort city located in San Diego, California. It's known for its luxury hotels, delicious restaurants, beautiful parks, and gorgeous beaches. It has just been announced that the…

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Learn About These Rare Eye Diseases to Honor Healthy Vision Month!

• Post author:Jean Martell
• Post published:May 20, 2019
• Post category:Rare Disease/Retinitis pigmentosa/Stargardt Disease/Stargardt disease 4/Usher syndrome- type 1F/Usher syndrome- type 2B

May is Healthy Vision Month! Rare disease or not, maintaining healthy eye health should be a priority for all of us. The National Institute of Health has resources for not…

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Experimental Treatment for Genetic Amyotrophic Lateral Sclerosis Shows Potential in Trial

• Post author:James Moore
• Post published:May 20, 2019
• Post category:Amyotrophic Lateral Sclerosis/Rare Disease

According to a story from BioSpace, the drug developer Biogen recently released interim results from a Phase 1/2 clinical trial. This clinical trial is testing the drug tofersen, which is…

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Join the 2019 Walk for Behcet’s Disease in Orlando, Florida for Awareness Day!

• Post author:Jean Martell
• Post published:May 17, 2019
• Post category:Behçet's/Rare Disease

Behcet’s Awareness Day is May 20th! Behcet’s disease (also known as Behçet’s and Behçet’s syndrome) is a rare condition that causes chronic inflammation which manifests as ulcers (commonly referred to…

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Living with Blepharospasm, Coping with Social Phobia

• Post author:Patient Worthy Contributor
• Post published:May 17, 2019
• Post category:Dystonia/Rare Disease

I was diagnosed with benign essential blepharospasm at the end of 2015. This is a neurological condition which causes the muscles around my eyes to contract, resulting in uncontrollable blinking…

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SomaLogic is Working to Improve Precision Medicine for Rare Diseases

• Post author:Trudy Horsting
• Post published:May 16, 2019
• Post category:Duchenne Muscular Dystrophy/Rare Disease

The Growth of Precision Medicine Precision medicine is the idea that each patient is an individual, and therefore deserves an individualized treatment plan to secure the best outcomes. It is…

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Experimental Treatment for Multiple Myeloma Earns Orphan Drug Designation

• Post author:James Moore
• Post published:May 16, 2019
• Post category:Multiple Myeloma/Rare Disease

According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…

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Editor’s Choice: Rare Patients Helping Other Patients

• Post author:Patient Worthy Contributor
• Post published:May 15, 2019
• Post category:Rare Disease

Happy Wednesday! We're officially halfway through May if you can believe it! This week we're highlighting a piece about an intern using their rare experience to inform their work, followed…

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An Experimental Treatment for Adenoid Cystic Carcinoma Earns Orphan Drug Designation

• Post author:James Moore
• Post published:May 15, 2019
• Post category:Adenoid Cystic Carcinoma/Rare Disease

According to a story from apnews.com, the drug developer Ayala Pharmaceuticals has recently announced that the company's investigational product candidate, known as as AL101, has earned Orphan Drug Designation. This…

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NIH Reverses Decision, Allows Doctors to Speak to Federal Investigators About Sepsis Study Objections

• Post author:Scott Carlson
• Post published:May 14, 2019
• Post category:Rare Disease

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…

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New Bill Could Allow Easier Access to Complex Rehabilitation Technology for Rare Disease Patients

• Post author:Trudy Horsting
• Post published:May 14, 2019
• Post category:Cerebral Palsy/Multiple Sclerosis/Muscular Dystrophy/Rare Disease

H.R. 2408 H.R. 2408 is a new piece of federal legislation otherwise known as the "Ensuring Access to Quality Complex Rehabilitation Technology Act." Essentially, this act creates a new category…

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The Chronically Ill and Disabled Deserve Better Than Discrimination and Harassment from the TSA

• Post author:James Moore
• Post published:May 14, 2019
• Post category:Rare Disease

David Rowland, age 77, is a cancer survivor and former veteran of the Air Force. He is also a tube feeder. People who use a feeding tube are considered disabled…

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Boy with Scleroderma has Donated Over 7,000 Gifts to Other Children in the Hospital

• Post author:Trudy Horsting
• Post published:May 14, 2019
• Post category:Rare Disease/Systemic Scleroderma/Systemic Sclerosis

Scleroderma Scleroderma refers to a whole group of diseases, all rare, that cause the skin and connective tissues in the body to harden. This is a result of excess collagen…

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