Free MDS Foundation Forum in MD!
Patient Worthy's partner the MDS Foundation is hosting a MDS Patient & Family/Caregiver Forum Baltimore, Maryland in conjunction with the University of Maryland Marlene and Stewart Greenebaum Cancer Center. This…
Drink One For Dane: Dutch Bros Coffee Hosts 13th Annual Amyotrophic Lateral Sclerosis Fundraiser This Friday, May 10th
Dutch Bros Coffee is hosting their 13th annual fundraiser for amyotrophic lateral sclerosis (ALS) this Friday, May 10th, 2019. It's called "Drink One for Dane" in honor of Dane Boersma,…
ISMRD International Conference for Glycoprotein Storage Diseases Coming This July
Upcoming Conference The International Society for Mannosidosis & Related Diseases (ISMRD) serves as an international patient advocate for those living with glycoprotein storage diseases. These include alpha-mannosidosis, fucosidosis, mucolipidosis type…
Upcoming FREE MDS Event in PA!
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. When: Saturday, April 27, 2019 Where: West Penn Hospital 4800 Friendship Avenue Pittsburgh, PA 15224 Wintergarden Conference Center – First…
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All-New Canadian Rare Disease Research Platform Begins Operations
According to a story from Gene42, a new rare disease platform called Genomics4RD has been launched in Canada. This is the first national research platform dedicated to rare disease to…
RARE2019 Conference to Take Place this Week in Austria
Second Annual International Congress on Advanced Treatments in Rare Diseases The first International Congress on Advanced Treatments in Rare Diseases, otherwise known as RARE2018, was held in London, England. Now,…
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First Treatment for aTTP Approved by FDA
According to a story from Pharmacy Times, the US Food and Drug Administration (FDA) have recently approved the first ever therapy for the rare disorder acquired thrombotic thrombocytopenic purpura (aTTP),…
Don’t Miss The Final Screening of “Take A Look At This Heart,” About Love and Intimacy in the Disabled Community
A rarely addressed subject that comes up in discussions about people with disabilities is the subject of romantic relationships, intimacy, and parenting. More than likely, a lot of day to…
Huntington’s and Hope: Seth’s Story, Part 2
Check out part one of Seth's story here. Seth had set a google alert for his hometown, Natick, to stay up to date in his home's local news. One day,…
Father Gets Tattoos of Son’s Scars as Symbol of Eternal Support
This wonderful story was brought to Patient Worthy by our partners, the Cystinosis Research Network (CRN). The season of giving started off in an unconventional setting for the Moore Family. Chandler…
Huntington’s and Hope: Seth’s Story, Part 1
When Seth was twelve years old, he and the rest of his family began to realize something was wrong with his mother. Seth can remember conversing with her pleasantly, and…
This Girl’s Diffuse Intrinsic Pontine Glioma Tumor is Gone, But No One Knows Why
According to a story from CBS News, Roxli Doss, an eleven year old girl, was diagnosed with a rare and dangerous brain tumor called diffuse intrinsic pontine glioma. It is…
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Vote to Help Family Win 50,000 Dollars for Mitochondrial Disease Research!
There are only 11 people in the world diagnosed with Mitochondrial Complex 1 Deficiency. Katherine Faughn, a 7-year-old living in Kentucky is one of these 11. The disease wasn't even…
<a href="https://pixabay.com/users/Tabeajaichhalt/">Tabeajaichhalt</a> / Pixabay
ICYMI: Creator of “SpongeBob SquarePants” Dies After Being Diagnosed With Amyotrophic Lateral Sclerosis
According to a story from Buzzfeed News, Stephen Hillenburg, the creator of the smash hit children's cartoon SpongeBob SquarePants has passed away at age 57. Last year, Hillenburg had revealed…
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UK Cystic Fibrosis Patients are Protesting to get a Vital Drug Covered on the NHS
Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…
Test Results Offer New Hope to Ankylosing Spondylitis Patients
According to a recent press release, patients living with ankylosing spondylitis may soon have another treatment option. The researchers studying the new treatment, known as ixekizumab, recently posted results from…
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Want to Learn More About Clinical Trials? Don’t Miss This Free Webinar
The clinical research process is a thorough and time consuming method in which new drugs, therapies, and medical devices are rigorously tested before being made available to the public. Through…
Young Advocate Speaks at UN for Children with Psoriatic Arthritis
At 14 years old, Bede Marciari has experienced and missed out on a great number of things. For many people, this is just part of growing up. In Bede’s case,…
<a href="https://pixabay.com/users/Free-Photos/">Free-Photos</a> / Pixabay
Energy Walk for Life Sets Mitochondrial Disease Advocacy in Motion
Linda Ramsey had a son named Kevin. In the beginning everything was okay, and Kevin grew normally for a short time. After a few months, his experienced difficulty swallowing. He…
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Data Looks Good For Synlogic’s Experimental Phenylketonuria Drug, And If You’re a Patient, You Can Get Involved
The biotechnology company Synlogic announced positive data from its most recent Phase 1/2a clinical trial of its experimental product SYNB1618. This trial did not test the therapeutic action of the…
<a href="https://pixabay.com/users/Mysticsartdesign/">Mysticsartdesign</a> / Pixabay
The First Treatment for Neurotrophic Keratitis Was Just Approved
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently approved the drug Oxervate (cenegermin). This treatment is the first FDA approved therapy for the rare…
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New Treatment for Fabry Disease Gets Cleared by the FDA
According to a story from MD Magazine, the US Food and Drug Administration (FDA) has approved the medication migalastat, also known as Galafold, for the treatment of Fabry disease. This…
<a href="https://pixabay.com/users/cherylholt/">cherylholt</a> / Pixabay
Cystic Fibrosis Drug ORKAMBI Now Approved for Patients Age 2-5 by the FDA
According to a story from BioSpace, an announcement from the biotechnology company Vertex Pharmaceuticals revealed that the U.S. Food and Drug Administration has approved an expanded indication for ORKAMBI, the…
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PatientWorthy is Offering Two Tickets to the First Annual Alagille Syndrome Alliance Gala
PatientWorthy will be offering a pair of tickets to the Alagille Syndrome Alliance's (ALGSA) First Annual Gala. The tickets will be available for patients or caregivers in the San Diego…
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Express Scripts Makes Diagnosis Easier with Second Opinion
According to PRNewswire, Express Scripts announced a new program to benefit people living with rare diseases on May 31st. The program, called Second Opinion is part of the company’s larger…
