Chip Wilson, billionaire founder of Lululemon Athletica, was first diagnosed with muscular dystrophy in 1987 at the age of 32. Wilson, who is now 66 years old told Global…
This year, the American Society of Gene & Cell Therapy (ASGCT) held its 26th annual meeting from May 16 through May 20, 2023. In a news release, biotechnology company…
A January 2023 news release from biopharmaceutical company Avidity Biosciences, Inc. ("Avidity") shares that the company's therapy, AOC 1020, earned Fast Track designation from the FDA for the treatment of…
Actor Max Adler is known for a variety of roles in films and TV shows such as Glee, Saugatuck Cures, and Switched at Birth. But did you know that…
As part of its 2022 webinar series, the Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP) hosted a webinar on August 17, 2022 titled "Addressing the Gaps in Clinical…
In a news release, biopharmaceutical company Fulcrum Therapeutics, Inc. ("Fulcrum") shared that the first patient was dosed in the Phase 3 REACH study. Within the study, researchers are evaluating…
Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
Genetic editing has the potential to address and treat a variety of genetic conditions. Recently, researchers explored gene editing as a potential therapeutic option for patients with facioscapulohumeral muscular dystrophy…
Ray Jordan and Cyndi Segroves were born thousands of miles away from one another; in fact, they grew up in different countries. Cyndi is from the US and Ray is…
Colette Wheeler is one of four siblings, and although each had only a 50/50 chance of inheriting the genetic mutation that leads to facioscapulohumeral muscular dystrophy (FSHD), all of them…
by Danielle Bradshaw from In The Cloud Copy Fulcrum Therapeutics, a company that focuses on clinical-stage biopharmaceuticals to improve the quality of life for patients that have rare genetic diseases,…
A team of researchers from the University of Alberta have created a possible treatment for facioscapulohumeral muscular dystrophy (FSHD). Their work has been published in the Proceedings of the National Academy…
According to a press release from the Boston Children's Hospital, a study has discovered the efficacy of CRISPR-Cas9 gene-editing technology in the treatment of facioscapulohumeral muscular dystrophy (FSHD). As…
Losmapimod is an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD), and it recently received the Orphan Drug designation from the FDA. Not only did it receive this designation, but its…
A recent press release from Fulcrum Therapeutics, a Massachusetts-based biotechnology company, announced preliminary findings of the company's phase 1 clinical study of losmapimod in the treatment of facioscapulohumeral dystrophy. Losmapimod is…
The Bad News Unfortunately, it has recently been announced that an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD) will not be continuing on in clinical trials. This is because the…
It was announced this Monday that Fulcrum Therapeutics, a Phase 1 biotech developing therapies based on gene regulation for rare diseases, announced terms for its initial public offering (IPO), aka its…
According to a press release from Massachusetts-based biotech company Fulcrum Therapeutics, the company has successfully secured global commercialization rights of GlaxoSmithKline-developed experimental facioscapulohumeral muscular dystrophy (FSHD) drug losmapimod. Losmapimod, an…
According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration…
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