Patient Worthy partner HCU Network America and RARE X, a rare disease patient data service organization, have recently partnered in order to create a registry of patients living with homocystinuria,…
According to a story from pharmaphorum.com, a therapy being developed for the rare disease activated PI3K delta syndrome (APDS) is on its way to regulators for approval following promising results…
Jamas and Margot LaFreniere started the Sophie's Hope Foundation in 2020 shortly after Sophie, their daughter, was diagnosed with a rare disease: glycogen storage disease type 1B (GSD1B). The mission…
According to a story from Biopharma Dive, the US Food and Drug Administration (FDA) has recently approved a treatment for a rare form of melanoma that affects the eyes, called…
This story is brought to you by our friends at HAE Junior in the Czech Republic. I think that the best gift is one that will please not only the…
According to a story from PR Newswire, Myeloma Action Month takes place every year during the month of March. This year, the International Myeloma Foundation (IMF) is organizing around the…
Written By Katie Ward I am not one who likes to bring attention to themselves, and you won’t ever find me talking about my struggles, issues, or anything negative. I…
On February 23, 2022, the National Organization for Rare Disorders (NORD) held a webinar titled "Advancing Equity in Rare Healthcare." The program was organized in collaboration with the Rare Disease…
A recent study examined the impact of age on clinical outcomes for patients diagnosed with chronic lymphocytic leukemia (CLL). This study was published in the journal Blood Advances. All patients in…
Extensive sweating, or hyperhidrosis, can be an extremely frustrating diagnosis. Although sweating itself is an incredibly normal and healthy process, when it happens excessively, it can impact your day to…
According to a story from the Baltimore Sun, a childhood home of Henrietta Lacks - a Black woman often dubbed "the mother of modern medicine" - was unknowingly demolished just…
A recent study has uncovered that a CAR-T therapy called Kymriah could lead to improved results for follicular lymphoma patients. Specifically, it was found to be both effective and safe…
Rare Disease Week 2022 is taking place this year on February 22nd to March 2nd. Though normally held in Washington, DC, the EveryLife Foundation for Rare Diseases, which organizes the…
Jordan's Guardian Angels, a nonprofit organization that supports the Jordan's syndrome community, has recently announced a new scholarship that will honor children that have died as a result of being…
On January 27, 2022, The EveryLife Foundation for Rare Diseases, in partnership with the Biotechnology Innovation Organization (BIO), National Health Council, and Pharmaceutical Research and Manufacturers of America (PhRMA), hosted…
According to a press release from the pharmaceutical company Bristol Myers Squibb, the company's CAR T-Cell therapy idecabtagene vicleucel (marketed as Abecma) has been approved by the Japanese Ministry of…
The World Health Organization (the WHO) announced World Leprosy Day which this year will be celebrated on January 30th. The theme of the celebration is “United for Dignity.” It…
On January 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted its first monthly webinar for 2022. In this month's program, the speakers provided an overview of the progress that…
Moebius Syndrome Awareness Day is January 24th! This event was created in 2011 by the Many Faces of Moebius Syndrome (MFOMS). It is now celebrated across the world, as the…
Recently, Chain Drug Review reported that the FDA had approved a generic form of colchicine pills (normally prescribed as Mitigare, Colcrys, or Gloperba) for the treatment of familial Mediterranean fever (FMF).…
PTC Therapeutics has just announced that their therapy Waylivra (volanesorsen), a treatment for familial chylomicronemia syndrome (FCS), has been given Category 1 classification from the Drug Market Regulation Chamber in…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
Saniona has just announced that their Phase 2b clinical trial examining Tesomet as a treatment for Prader-Willi syndrome (PWS) will be beginning soon. This treatment is a combination therapy which…
The recent FDA approval of Vyvgart (efgartigimod) for generalized myasthenia gravis (gMG) marks the first drug approval for Argenx. Vyvgart is designed specifically for adults who test positive for…
Three organizations focused on amytrophic lateral sclerosis (ALS) as well as other rare neurodegenerative diseases, have just celebrated the House of Representatives' passage of the ACT for ALS (H.R.3537/S.1813). The…
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