AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina. This company is dedicated to the research of gene therapies as potential therapeutic options…
According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…
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…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…
PTC Therapeutics is a pharmaceutical company that has been working with Translarna, a medication intended for the treatment of Duchenne muscular dystrophy (DMD). They analyzed this treatment and presented the…
According to a story from newsroom.heart.org, one of the most substantial domestic policy achievements of the Obama administration was the passage of the Affordable Care Act (ACA). Since its passage,…
More than 20 years ago researchers at Harvard University, led by professor Oliver Pourquié, discovered how the vertebra in chickens first form. Essentially, a 'tick' initiates the formation of a…
According to an announcement from the Muscular Dystrophy Association, the organization has recently awarded a total of $1,076,232 in MDA Venture Philanthropy (MVP) funding towards the drug company AavantiBio, which…
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved the drug golodirsen (marketed as Vyondys 53) as a treatment for Duchenne muscular dystrophy…
A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…
According to a story from tradeshownews.vporoom.com, the Muscular Dystrophy Association (MDA) has recently announced the official approval of its Patient-Focused Drug Development (PFDD) meeting by the US Food and Drug…
Researchers at the University of Pennsylvania are working on a new potential gene therapy for Duchenne muscular dystrophy (DMD). DMD is a rare form of muscular dystrophy that primarily affects…
According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…
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…
According to a story from BioSpace, a global collaborative called the Trajectory Analysis Project recently released a study that utilized the North Star Ambulatory Assessment (NSAA), a recently developed measurement…
According to a story from 10tv.com, Kelly Maynard could be hear cheering loudly from the sidelines as her son Jackson, aged 12, scored a touchdown in one of his middle…
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…
According to a story from Financial Buzz, the Steadman Philippon Research Institute (SPRI) recently received an award in research from the National Institutes of Health (NIH). The award is in…
According to a publication from NewZNew, the NeuroGen Brain and Spine Institute in India plans on hosting an outpatient stem cell therapy consultation camp in Chandigarh on the last day…
According to a story from Xconomy, the US Food and Drug Administration (FDA) made a controversial decision three years ago when it approved the drug eteplirsen as a treatment for…
According to a story from BioPortfolio, the drug company Pfizer has recently presented data from its phase 1b clinical trial that tested the company's experimental gene therapy PF-06939926, which is…
.jpg)
The first indication of the influence that people can have in clinical trials when acting as a community may have occurred in the 1980s. As reported recently in Viral…
According to a story from blog.cincinnatichildrens.org, a diagnosis of Duchenne muscular dystrophy is always an alarming piece of news; learning that your child has a progressive muscle atrophy disorder that…
According to a story from Science Daily, a recent study has found that lithium chloride was able to improve muscle size and strength in a mouse mode of a specific…
For most of us, our body follows a predictable timeline when it comes to growing older. While there's some variation depending on genes and lifestyle, each year, we age with…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
