August 2019 - Patient Worthy

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

Post author:Scott Carlson
Post published:August 30, 2019
Post category:Pancreatic Cancer Rare Disease

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

Post author:Scott Carlson
Post published:August 30, 2019
Post category:Batten Disease Fabry Disease Pompe Disease

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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New Progress is Being Made Against Often Overlooked Tropical Diseases

Post author:James Moore
Post published:August 30, 2019
Post category:Rare Disease

According to a story from New Statesman, the last month has seen a number of new drugs introduced that could have a significant impact on several dangerous tropical diseases, such…

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First Human Trial Completed for Experimental Scleroderma and Multiple Sclerosis Drug

Post author:James Moore
Post published:August 30, 2019
Post category:Multiple Sclerosis Systemic Scleroderma Systemic Sclerosis

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

Post author:Scott Carlson
Post published:August 30, 2019
Post category:Systemic Sclerosis

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…

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

Post author:Scott Carlson
Post published:August 30, 2019
Post category:Niemann-Pick Type C Disease

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…

Former NFL Star Tim Green has ALS but No Regrets About His Career

Post author:Rose Duesterwald
Post published:August 30, 2019
Post category:Amyotrophic Lateral Sclerosis

In 1996, Tim Green made the comment that you cannot go through an NFL season without doing serious damage to your body. In a recent 60 Minutes interview with Steve…

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Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation

Post author:James Moore
Post published:August 30, 2019
Post category:Myelodysplastic syndromes Pancreatic Cancer Rare Disease

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…

Babies in Spain Develop Rare ‘Werewolf Syndrome’ After Contamination

Post author:Jean Martell
Post published:August 29, 2019
Post category:Hypertrichosis lanuginosa- acquired

According to a report by the Spanish newspaper El País, over a dozen children in Spain have recently been diagnosed with the rare condition hypertrichosis -- also known as werewolf…

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The FDA is Finally Implementing Rules for Fecal Transplants—But Someone Had to Die First

Post author:James Moore
Post published:August 29, 2019
Post category:Rare Disease

According to a story from NBC News, the death of a patient following a fecal transplant procedure has finally prompted the US Food and Drug Administration (FDA) to take more…

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

Post author:Trudy Horsting
Post published:August 29, 2019
Post category:Achalasia

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

Post author:James Moore
Post published:August 29, 2019
Post category:Pantothenate kinase-associated neurodegenerat

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…

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

Post author:Trudy Horsting
Post published:August 29, 2019
Post category:MPS VI (Maroteaux-Lamy syndrome)Mucopolysaccharidosis

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…

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

Post author:Rose Duesterwald
Post published:August 29, 2019
Post category:Amyotrophic Lateral Sclerosis

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

Post author:James Moore
Post published:August 29, 2019
Post category: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…

A Hereditary Angiodema Story: Is it Okay for Me To Laugh and Smile?

Post author:Patient Worthy Contributor
Post published:August 29, 2019
Post category:Hereditary Angiodema Rare Disease

As my children are getting older they seem to create elaborate stories of why they didn’t clean up their room or complete any other task on their to-do list. I’ve…

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Medical Student Keeps Pursuing Her Dream with CIDP and CRPS

Post author:James Moore
Post published:August 28, 2019
Post category:Chronic Inflammatory Demyelinating Polyneuropathy Complex Regional Pain Syndrome

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.…

Mystery: A Paralysis That Occurs in Children Every-Other-Year

Post author:Rose Duesterwald
Post published:August 28, 2019
Post category:Acute flaccid myelitis West Nile virus Zika Virus

The Center for Disease Control (CDC) is urging doctors and researchers to investigate and to collect new data before the disease strikes again. An article in Ars Technica reports…

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

Post author:James Moore
Post published:August 28, 2019
Post category:Spinal Muscular Atrophy

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

Post author:Scott Carlson
Post published:August 28, 2019
Post category:ulcerative Colitis

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

Post author:Rose Duesterwald
Post published:August 28, 2019
Post category:Anaplastic Lymphoma Kinase-Positive Non-Small Cell Lung Cancer Cancer

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

Post author:James Moore
Post published:August 28, 2019
Post category:Mucopolysaccharidosis Sanfilippo Syndrome

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)…

FDA Gives Accelerated Approval to Drug Targeting NTRK and ROS1 Mutated Cancers

Post author:Scott Carlson
Post published:August 27, 2019
Post category:Cancer

According to a publication from CURE Today, the US Food and Drug Administration (FDA) recently approved Genentech, Inc's Rozlytrek (generic name entrectinib) for the treatment of cancer patients who have…

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Experimental Treatment for Immune Thrombocytopenia Begins Trials in China

Post author:James Moore
Post published:August 27, 2019
Post category:Immune thrombocytopenia Thrombocytopenia

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

Post author:James Moore
Post published:August 27, 2019
Post category:Rare Disease

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…

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

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

New Progress is Being Made Against Often Overlooked Tropical Diseases

First Human Trial Completed for Experimental Scleroderma and Multiple Sclerosis Drug

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

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

Former NFL Star Tim Green has ALS but No Regrets About His Career

Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation

Babies in Spain Develop Rare ‘Werewolf Syndrome’ After Contamination

The FDA is Finally Implementing Rules for Fecal Transplants—But Someone Had to Die First

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

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

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

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

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

A Hereditary Angiodema Story: Is it Okay for Me To Laugh and Smile?

Medical Student Keeps Pursuing Her Dream with CIDP and CRPS

Mystery: A Paralysis That Occurs in Children Every-Other-Year

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

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

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

IND Accepted for an Experimental Sanfilippo Syndrome Treatment

FDA Gives Accelerated Approval to Drug Targeting NTRK and ROS1 Mutated Cancers

Experimental Treatment for Immune Thrombocytopenia Begins Trials in China

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

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

“Shades of Psoriasis”: Plaque Psoriasis and Sharing Your Story

Understanding RSV: Why Combatting Ethnic and Age-Related Health Disparities Could Improve Outcomes

We Need Equity and Diversity Within Rare Disease Research

DNA in Color: How Ronya Nelson is Working to Close the Diversity Gap in Genetics - Part 1

Bridging the Diversity Gap in Healthcare

Congratulations to the 2023 Recipients of the Diversity in Lupus Research Awards!

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