Recently, pharmaceutical company RIBOMIC announced that the first patient was dosed in their Phase 1 clinical trial. Ultimately, the trial is meant to determine whether RBM-007 could be an effective…
For a lot of us, self-care is difficult. This is for a lot of reasons, some of which might be guilt, not enough time, putting others before ourselves, not sure…
The 2020 Living Rare, Living Stronger NORD Patient and Family Forum July 18-20, 2020 Cleveland, OH This yearly conference for patients, their families, and researchers features a comprehensive content program…
According to a story from Fabry Disease News, any member of the Fabry disease patient community should strongly consider signing up for the Fabry Registry. Getting involved comes with several…
According to a story from proactiveinvestor.com.au, the drug company Pharmaxis Ltd. announced recently that the US Food and Drug Administration (FDA) has granted it Orphan Drug designation. This designation is…
According to a story from Fabry Disease News, a recent case study analyzed the situation of two cousins living with Fabry disease who were being treated with enzyme replacement therapy,…
According to a story from Star Tribune, Chelsea Coenraads was 14 months old when her mother Monica became convinced that something seriously wrong with her daughter. She had failed to…
According to Ankylosing Spondylitis News, a new treatment, Hulio, has been approved by the FDA. It is a treatment for ankylosing spondylitis and other inflammatory disorders. Although it has been…
According to a story from Korea Biomedical Review, Seoul National University Hospital (SNUH) in South Korea has just announced recently that the process of recruiting patients for the National Bio…
Living with a rare disease can come with some difficulties, whether those are physical, mental, or financial. It is estimated that 30 million Americans live with a rare disease, a…
By Lauren Taylor from In The Cloud Copy Biogen announced new data from the NURTURE trial of pre-symptomatic patients with spinal muscular atrophy (SMA). SMA is a rare, autosomal recessive…
According to a story from keloland.com, Westin Cuka, age six, was first diagnosed with neurofibromatosis, a rare disorder, three years ago. Since then, the family has traveled on multiple occasions…
Rare pediatric disease designation has just been granted to Rhythm Pharmaceuticals for their investigative therapy called setmelanotide. This drug is being investigated as a therapeutic option for two conditions- leptin…
In a July 9 press release, the Familial Chylomicronemia Syndrome (FCS) Foundation announced its support for a bill introduced by Congressman Paul Tonko (D-NY) and David B. McKinley (R-WV).…
Each year, June 13th is celebrated as Gastrointestinal Stromal Tumors (GIST) Awareness Day. The ultimate goal of awareness days for rare diseases is to help inform both the general public…
Alaina Johns, a Philadelphia based freelance writer, wears two hats. One hat as editor-in-chief of the Broad Street Review, and the other as a patient with several chronic illnesses.…
According to a story from GlobeNewswire, the biopharmaceutical company Onconova Therapeutics, Inc. has recently published results from a phase 1 clinical trial that was sponsored by the company. This trial…
Data has recently been published to support a new diagnostic method for progressive supranuclear palsy, according to GlobeNewswire. Life Molecular Imaging and AC Immune SA have reported this data, which…
By Jodee Redmond from In The Cloud Copy When a patient identified as Ms. B in a case history complained of headaches starting at the back of her head and…
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…
July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
Timber Pharmaceuticals focuses on rare diseases of dermatologic origin. These include scleroderma, congenital ichthyosis, tuberous sclerosis complex, and facial angiofibromas. They are currently working on two clinical trials. Trial 1…
CMT1A Charcot-Marie-Tooth disease (CMT) has a few different subtypes. 60% of all patients diagnosed with type 1 of the rare disease have CMT1A. This subtype means patients have an duplicate copy…
According to a recent press release, EspeRare received Breakthrough Therapy Designation for its investigational therapy ER-004, designed for the prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED). Ultimately, this…
According to ASH Clinical News, a phase 1 trial of lenzilumab illustrated clinical benefits for approximately one third of participants with chronic myelomonocytic leukemia. It is a novel antibody, and…
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