Maybe you remember the day of your diagnosis: shocking, confusing, chaotic. Realizing the news will change your life, but not quite knowing how. Jessie Ace was 22 years old, finishing…
As originally reported in Cambridge Independent, inflammatory bowel diseases are about to get a boost in research after Health Data Research UK has awarded a £5 million grant to a…
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Waukesha Wisconsin Police Captain Dan Baumann recently held a news conference that was described in an article published in Scientific American. Captain Baumann said that parents of a teenage boy showed…
A recent publication from the US Food and Drug Administration announced the regulatory authority's approval of Nourianz (generic name istradefylline) for Parkinson's patients during "off" episodes. "Off" episodes are windows…
The Center for Chronic Illness is a nonprofit group that is based out of Seattle, Washington. The goals of this organization include the provision of support and educational resources to…
According to a story from Yahoo! News, Dorothy Dunnington is beginning to wonder if she will ever fully understand the circumstances surrounding the death of her sister Chrissy, who was…
According to a story from Belfast Live, parents Tara and Glenn Blaine didn't take long to recognize that their newborn daughter Mia was not developing normally. Ultimately, the little girl…
With advocacy organizations gaining traction, sometimes patients with rare diseases may have a handful of options to choose from. Some patients may be quick to join patient advocacy groups…
According to a story from The Guardian, Jeremy Corbyn, the leader of the UK Labour Party, announced a remarkable plan at the most recent Labour party conference that has the…
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…
Innovation and Collaboration Macular telangiectasia type 2 (MacTel) is a rare eye disease that causes progressive vision loss as patients age. Paul Bernstein is a physician at the John A.…
The regenerative medicine market is witnessing a high growth rate across the world. The increasing incidence of bone injuries and joint fractures are boosting this market. According to Transparency Market…
According to a story from Financial Buzz, the biotherapeutics company Bellerophon Therapeutics Inc. has recently announced that it has earned Orphan Drug designation from the US Food and Drug Administration…
Consent is a concept back under discussion in modern conversations as we readdress what it means to cross the line. Consent is no longer seen in the black and white…
According to a story from Fabry Disease News, the first patient in a clinical trial testing the investigational gene therapy FLT190 has reportedly been dosed. FLT190 is being developed as…
According to a story from finanznachrichten.de, the latest data from three phase 3 studies is giving further confirmation to the capability of the recently approved gene therapy Zolgensma to have…
According to a story from Spectrum News a recent study revealed two potential therapies that could treat symptoms associated with Rett syndrome, a rare disorder. The study involved the creation…
According to a study recently published in Allergy and Asthma Proceedings, certain risk factors may increase an individual's likelihood of receiving a late diagnosis for hereditary angioedema. Hereditary angioedema is…
Cystinosis Cystinosis is a rare disease that causes cystine, an amino acid, to build up in the body's organs. However, there is treatment that can prevent this accumulation. Normally, patients…
Ischemic cardiomyopathy is a type of dilated cardiomyopathy, in which the capability of heart to pump blood is reduced. This causes lack of blood supply to heart muscles which result…
Happy Thursday everyone! This week, we're sharing a video that will warm your heart, as well as an article about how to improve care in older Multiple Myeloma patients. We…
When I was 12 years old, following my mom’s sudden cardiac arrests, I was diagnosed with a rare, genetic sudden arrhythmia death syndrome called Congenital Long QT Syndrome Type 5.…
In a story from Healio, Dr. Niraj C. Patel, who is an associate professor of pediatric infectious disease and immunology at Levine Children’s Hospital, Atrium Health in Charlotte, NC, is…
According to a story from medicalxpress.com, Dr. Nathan Hoot is an emergency medical physician, but his role as a doctor doesn't entirely end when he leaves the hospital because his…
According to the American Journal of Managed Care (AJMC), the US Food and Drug Administration (USFDA) has approved Pexidartinib (Turalio) drug as first targeted therapy to treat symptomatic Tenosynovial Giant…
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