One of our partners, the MDS Foundation, Inc, is sponsoring another free MDS Patient & Family/Caregiver Forum. This event will be held in Birmingham, Alabama When: Saturday, February 1, 2020…
One of our partners, the MDS Foundation, Inc, is sponsoring another free MDS Patient & Family/Caregiver Forum. This event will be held in Houston, Texas When: Saturday, February 22, 2020 Where:…
I wanted to invite the invisibly disabled community to join the screening of ‘We Are Visible’, my film about people living with an invisible condition (Ehlers-Danlos syndrome) all around the…
A press release from the US Food and Drug Administration (FDA) is announcing the approval of a new treatment for anemia in adult patients with the rare blood disorder beta…
Tay-Sachs Disease September is Tay-Sachs Disease Awareness Month. Voted on by the Senate unanimously in 2008, this month aims to spread awareness about this disease and the importance of carrier…
According to a story from finanzen.at, the drug nintedanib (marketed as Ofev) has officially been approved by the US Food and Drug Administration (FDA) as a treatment for interstitial lung…
Cure GM1 Foundation is hosting a family meeting filled with hope on Friday, September 20th! This gathering will be focused on community, clinical development and research updates, patient advocacy, and…
According to a story from fda.gov, the US Food and Drug Administration (FDA) has approved the drug pexidartinib (marketed as Turalio) as a treatment for tenosynovial giant cell tumor (TGCT)…
According to a story from globenewswire.com, the drug company Amicus Therapeutics recently announced that Argentina's Agency of Medicines, Food, and Medical Devices (ANMAT) has granted approval for the drug migalastat…
I had the pleasure of attending the Glut1 Deficiency Foundation’s 2019 Conference. While this article can't possibly do the event justice and encompass all it had to offer, we will…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. The event will take place in Seattle, Washington on August 10th, 2019. When: Saturday, August 10,…
Emily Ventura has never met in-person another person living with progressive familial intrahepatic cholestasis (PFIC), her daughter’s life-threatening ultra-rare genetic disease. That will change on June 21st when Emily and…
According to a story from PR Newswire, the Novartis company AveXis recently announced that the US Food and Drug Administration (FDA) has approved Zolgensma, making it the first ever gene…
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…
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,…
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…
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…
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…
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,…
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),…
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…
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,…
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…
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…
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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