When Yang Siyang learned his eight-year-old Lillian has the rare childhood cancer acute myeloid leukemia, he felt overwhelmed by shock, grief, uncertainty. There are many intense and complicated emotions that…
As reported in World Pharmaa Today, Valneva SE is a specialty vaccine company in the business of the creation and distribution of prophylactic vaccines that treat infectious diseases with serious…
As reported in Times Union; this was a year where the numbers of many contagious diseases saw steep declines as the world stepped up in handwashing and mask-wearing. In the…
Spinal muscular atrophy is rare progressive muscular disease that over time, erodes a patient’s movement. Without early intervention, the condition erodes the young patients ability to breathe and even swallow.…
As reported in PR NewsWire; a pivotal Phase II/III trial to create a therapy for activated PI3K delta syndrome (APDS) has announced they’ve successfully completed patient enrollment for a new…
As reported in I News; scientists may have found a cure for a devastating and fatal brain disease, and the world of rare brain diseases may never be the same.…
My nickname is Arti and I'm 11 years old. Because of my HAE diagnosis, I've been suffering from frequent and painful swellings since I was little. My life is divided…
As reported in Fierce Pharma; BridgeBio has earned FDA approval for their new targeted cancer treatment, Truseltiq. The approval is the biotech company’s first ever approval for a cancer drug,…
As reported in Physicians Weekly; a new study wanted to further characterize Enthesitis-related arthritis (ERA), a subtype of juvenile idiopathic arthritis (JIA) that is often is accompanied by anterior uveitis,…
As seen in a press release by Bioindustry; the UK BioIndustry Association (BIA) has published findings from a survey on the public’s interest in supporting rare access to affordable medicine…
As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of…
As reported in Charcot-Marie Tooth News; this year, Eurordis has kicked off a campaign to make sure those with rare diseases don’t get left behind in the coming century. Small…
As reported in PR Newswire, Alcyone Therapeutics is launching a new set of precise gene therapies for CNS disorders with the help of a new $23 million investment from RTW…
As seen in PR Newswire, researchers at the biopharmaceutical company OxThera AB have spent 52 weeks studying 25 patients with primary hyperoxaluria (PH), a rare disease that progressively damages the…
In a press release by Parkinson's Canada; researchers in Canada wanted to unpack why some people first lose their sense of smell before developing Parkinson’s disease with age. While losing…
In a study from the University of New Mexico, researchers found the majority of patients with nausea report immediate relief after using cannabis, while 96% of the users reported nausea…
As reported in Biospace; the clinical-stage biotech company Nanoscope Therapeutics, Inc. has announced a resonating gene therapy success: all participants with advanced retinitis pigmentosa (RP) maintained vision improvements a year…
A press release by MYND Sciences announced that the drug research and development company, MYND Life Sciences Inc. has their new intellectual patent for their method of finding biomarkers to…
As COVID-19 makes its way around the globe, scientists are discovering new ways the infection will linger in the months and years to come. As reported in John Hopkins Medicine,…
As reported in Biospace, the biotech company Jasper Therapeutics Inc. has announced the start of a Phase 1/2 clinical trial on a potential conditioning agent, JSP191, to facilitate hematopoietic cell transplants,…
As the pandemic closed bars and theaters and so many of our jobs became remote, much of the world became virtual. And as cocktail hour moved onto zoom and museums…
Medscape reported that when Victoria Gray was three months old, her family was told to keep her close: her prognosis wasn't looking good. She had been rushed to the ER…
Though myalgic encephalomyelitis or chronic fatigue syndrome (CFS) is more widespread than many people realize, the condition has few resources and no remedy. The disabling condition is complex, with no…
As reported in News Wise, in Hackensack Meridian Jersey Shore University Medical Center and the Cleveland Clinic, one patient’s unlikely success story led researchers to conclude that a balance of…
As reported in the Northhampton Chronicle;, sometimes it's our toughest challenges that inspire us to grow taller than we thought possible and to push past our notions of our limits.…
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