2018 – Page 37 – Patient Worthy

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Takeda and Ovid Have Provided an Update on the Clinical Development Program for an Investigational Anti-Epileptic Drug

Post author:Anna Hewitt
Post published:August 21, 2018
Post category:CDKL5 Dravet Syndrome Dup15q Syndrome Lennox-Gastaut syndrome

Takeda Pharmaceutical Company Limited and Ovid Therapeutics have released an overview of the clinical development program for TAK-935/OV935, an investigational drug that is being developed as a potential anti-epileptic…

First of Its Kind Organization Offers In-Home Care for Kids with Spinal Muscular Atrophy

Post author:Athena Lee
Post published:August 21, 2018
Post category:Spinal Muscular Atrophy

According to the Santa Cruz Sentinel, Josalyn Catarino is an 11-year-old who loves Disneyland, TV romance shows, the ocean, and getting her fingernails painted. This, of course, sounds like any…

New Partnership May Have Implications for Those with Dravet, Alzheimer’s, Epilepsy, and More

Post author:Athena Lee
Post published:August 21, 2018
Post category:Dravet Syndrome

According to Newswire, the two companies, BioMotiv and Cure Network Dolby Acceleration Partners (CNDAP) partnered up together in order to create small molecules that can potentially treat various neurological disorders, including…

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FDA Grants Orphan Drug Designation to Onspira Therapeutics’ Investigational Interleukin-1 Receptor Antagonist for the Treatment of Bronchiolitis Obliterans

Post author:Patient Worthy Contributor
Post published:August 20, 2018
Post category:Rare Disease

According to a story from Business Wire, the biopharmaceutical company Onspira Therapeutics recently announced that its investigational therapy OSP-101 was recently granted Orphan Drug designation by the US Food and…

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Experimental Duchenne Muscular Dystrophy Drug Gets Multiple Designations From the FDA

Post author:James Moore
Post published:August 20, 2018
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy

According to a story from Globe Newswire, the biotechnology company Wave Life Sciences recently announced that its investigational product WVE-210201 has received both Rare Pediatric disease designation and Orphan Drug…

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The FDA Wants to Listen to Patient Voices in Medical Development and Regulation

Post author:Anna Hewitt
Post published:August 20, 2018
Post category:Rare Disease

The US Food and Drug Administration (FDA) has released a statement outlining its plans to promote patient voices in the development and regulation of medical products. You can find more…

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Warnings From the FDA go Largely Unnoticed by State Medical Boards

Post author:James Moore
Post published:August 20, 2018
Post category:Rare Disease

According to a story from USA Today, despite numerous warning letters sent from the US Food and Drug Administration to the offices of doctors and other medical professionals across the…

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Artificial Intelligence Can Accurately Recommend Patient Referrals For Eye Conditions

Post author:Anna Hewitt
Post published:August 20, 2018
Post category:Rare Disease

Researchers have found that an artificial intelligence (AI) system is able to recommend patient referrals for over fifty eye conditions as accurately as world-leading doctors. The project is part of…

For Low-Risk Thyroid Cancer, Doctors Shouldn’t Use the Word “Cancer”

Post author:Athena Lee
Post published:August 20, 2018
Post category:Thyroid Cancer

According to The Guardian, some researchers believe that the term "cancer" should be omitted from some medical diagnoses. Among these diagnoses include those for small thyroid cancer, low and intermediate…

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Planting the Seeds of Sarcoma Awareness

Post author:James Moore
Post published:August 20, 2018
Post category:Rare Disease Soft Tissue Sarcoma

According to a story from Deseret News, sarcoma patients at the Huntsman Cancer Institute are planting sunflowers as a way to spread awareness about sarcoma, a rare and aggressive form…

Important Questions to Ask After a Breast Cancer Diagnosis

Post author:Athena Lee
Post published:August 20, 2018
Post category:Metastatic Breast Cancer

According to the Dover Post, there are a few crucial questions to ask your doctor upon being diagnosed with breast cancer. By asking these crucial questions, patients will be…

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The FDA Has Approved the First Generic Version of EpiPen

Post author:Anna Hewitt
Post published:August 20, 2018
Post category:Rare Disease

The United States FDA has approved the first generic version of EpiPen and EpiPen Jr (epinephrine). The approval was granted to Teva Pharmaceuticals USA, who will now be able to…

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Two Companies are Collaborating to Develop an Investigational Drug for Epidermolysis Bullosa

Post author:Anna Hewitt
Post published:August 20, 2018
Post category:Epidermolysis Bullosa

Two companies, GtreeBNT and YuYang DNU, will collaborate in a joint venture to develop the investigational drug RGN-137 for the treatment of epidermolysis bullosa. For more details, you can read…

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Study Finds That BRAF Inhibitors Could be an Effective Treatment for Some Ovarian Cancer Patients

Post author:James Moore
Post published:August 18, 2018
Post category:Ovarian Cancer

According to a story from news-medical.net, a recent study suggests that a class of drugs called BRAF inhibitors could be an effective treatment option for ovarian cancer patients who carry…

The Burden of PKD: A Patient Story

Post author:[email protected]
Post published:August 17, 2018
Post category:Autosomal Dominant Polycystic Kidney Disease

If you or someone you know has ADPKD, email us at [email protected]. We'd love to hear from you about your experience! What is PKD and ADPKD? PKD (polycystic kidney disease)…

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So Why DOES Canada have Scarcer Access to Rare Disease Therapies?

Post author:James Moore
Post published:August 17, 2018
Post category:Cystic Fibrosis Fabry Disease Multiple Sclerosis Spinal Muscular Atrophy

According to a story from CheckOrphan, Durhane Wong-Rieger, the CEO of the Canadian Organization for Rare Disorders, says that the Canadian health system has been letting down the roughly three…

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Pediatric Cancer Rates are Highest in the Northeast, But No One Knows Why

Post author:James Moore
Post published:August 17, 2018
Post category:Brain Cancer

According to a story from nbcnews.com, a recent map from the Centers for Disease Control and Prevention (CDC) indicates that children have the highest rates of cancer in the Northeast.…

FREE MDS Event in VA Rescheduled for December 15th!

Post author:Rebekah
Post published:August 17, 2018
Post category:Myelodysplastic syndromes

Our partner, the MDS Foundation, is sponsoring another free event for patients with MDS and caregivers. Event Summary: Whether you are a newly diagnosed patient, a long-term survivor, or a caregiver…

Celebrate Children’s Health Day with Incredible and Free Resources from Sesame Street

Post author:Jean Martell
Post published:August 17, 2018
Post category:Rare Disease

Elmo and friends are here to help! - thanks to the good people of The National Association of Community Health Centers (NACHC)! For those who have never heard of the NACHC,…

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What Killed Aretha Franklin? An In-Depth Look at Pancreatic Neuroendocrine Tumors

Post author:James Moore
Post published:August 17, 2018
Post category:Neuroendocrine Tumors of Gastroenteric or Pancreatic Origin Pancreatic Cancer Rare Disease

On August 16th, 2018, Aretha Franklin, the "Queen of Soul," passed away at age 76. Aretha will be immortalized for generations because of the success of her classic soul ballads…

Teenager with Rare Disease Treks Across the Country with a Very Important Message

Post author:PW Collaborator
Post published:August 17, 2018
Post category:Primary Periodic Paralysis Rare Disease

Gabriel Low, a 17-year-old from Hawaii, just completed a 3,000-mile bike ride across the country. He’s been riding since June 11th when he embarked from Seattle, WA to raise awareness,…

How Getting Diagnosed With POTS Changed My Life: A Patient Story

Post author:Patient Worthy Contributor
Post published:August 17, 2018
Post category:Dysautonomia POTS Rare Disease

The Beginning My story began in February 2011. On January 10th, 2011, my husband and I were involved in an accident in which we slid off the side of a bridge 10 feet face…

Researchers May be Able to Predict Kidney Cancer Years in Advance

Post author:Anna Hewitt
Post published:August 17, 2018
Post category:Renal Cell Carcinoma

A recent study published in the journal Clinical Cancer Research has found that a certain protein called KIM-1 may be able to predict renal cell carcinoma up to five years before…

POTS and Vasovagal Syncope: A Patient Story

Post author:Patient Worthy Contributor
Post published:August 16, 2018
Post category:POTS Rare Disease

In January-March of 2015, I was the healthiest I’d ever been... or so I thought. I worked out, drank plenty of water, had a great lifestyle and meal plan. In…

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Alport Syndrome Patients Met With the FDA About Drug Development

Post author:James Moore
Post published:August 16, 2018
Post category:Alport syndrome Chronic Kidney Disease

According to a story from PR Newswire, patients with Alport syndrome, supported by the National Kidney Foundation and the Alport Syndrome Foundation, were involved in a meeting with representatives from…

Takeda and Ovid Have Provided an Update on the Clinical Development Program for an Investigational Anti-Epileptic Drug

First of Its Kind Organization Offers In-Home Care for Kids with Spinal Muscular Atrophy

New Partnership May Have Implications for Those with Dravet, Alzheimer’s, Epilepsy, and More

FDA Grants Orphan Drug Designation to Onspira Therapeutics’ Investigational Interleukin-1 Receptor Antagonist for the Treatment of Bronchiolitis Obliterans

Experimental Duchenne Muscular Dystrophy Drug Gets Multiple Designations From the FDA

The FDA Wants to Listen to Patient Voices in Medical Development and Regulation

Warnings From the FDA go Largely Unnoticed by State Medical Boards

Artificial Intelligence Can Accurately Recommend Patient Referrals For Eye Conditions

For Low-Risk Thyroid Cancer, Doctors Shouldn’t Use the Word “Cancer”

Planting the Seeds of Sarcoma Awareness

Important Questions to Ask After a Breast Cancer Diagnosis

The FDA Has Approved the First Generic Version of EpiPen

Two Companies are Collaborating to Develop an Investigational Drug for Epidermolysis Bullosa

Study Finds That BRAF Inhibitors Could be an Effective Treatment for Some Ovarian Cancer Patients

The Burden of PKD: A Patient Story

So Why DOES Canada have Scarcer Access to Rare Disease Therapies?

Pediatric Cancer Rates are Highest in the Northeast, But No One Knows Why

FREE MDS Event in VA Rescheduled for December 15th!

Celebrate Children’s Health Day with Incredible and Free Resources from Sesame Street

What Killed Aretha Franklin? An In-Depth Look at Pancreatic Neuroendocrine Tumors

Teenager with Rare Disease Treks Across the Country with a Very Important Message

How Getting Diagnosed With POTS Changed My Life: A Patient Story

Researchers May be Able to Predict Kidney Cancer Years in Advance

POTS and Vasovagal Syncope: A Patient Story

Alport Syndrome Patients Met With the FDA About Drug Development

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