The events of the past week-- and the past few hundred years-- have brought up a lot of sadness, anger, and reckoning in our country. There are so many ways…
A seemingly healthy baby was born in China. But as she grew up, her family started noticing that their daughter wasn't hitting motor milestones. She didn't start walking until she…
A few months ago, many of us were living completely different lives: going to rare disease conferences, heading to work each day, going out for food, seeing family and friends.…
Have you been feeling a little stiff or achy later? Maybe tired? I know that my body has undertaken a lot of stress thanks to the new life changes -…
The virtually-held National Kidney Foundation 2020 Spring Clinical Meetings explored various topics relating to nephrological conditions, treatment, and patient outcomes. One presentation, spearheaded by Dr. Adam Weinstein, MD, and…
Kelly and Rory McElligott, of Highton, Victoria, Australia, have 3 beautiful children: Maggie, Billy, and Alice. But the family was shocked when two of their children, Maggie and Billy,…
Recently, researchers from European Molecular Biology Laboratory (EMBL) looked to understand the cause of pulmonary arterial hypertension (PAH). They collaborated with Stanford University through the Life Sciences Alliance. Because…
Desde el distanciamiento social y los negocios cerrados hasta las preocupaciones personales de salud, COVID-19 ha cambiado la vida diaria en el futuro previsible. Hay casi 4.75 millones de diagnósticos…
In an article in Medical Xpress, Japanese doctors have performed a “first” by transplanting liver cells (made with embryonic stem cells) into a newborn. The six-day-old infant was diagnosed with…
As reported in Guru Focus, biotech company Protagonist Therapeutics has announced progression in their Phase 2 study on PTG-300 as treatment for patients with polycythemia vera, a rare cancer. Dr.…
According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…
By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…
Women can do anything that men can do, and vice versa. Well, while that sentiment may be true in our everyday life, it isn't quite true on a genetic…
IBDsmart Researchers at the University of Otago, led by Michael Shultz, have developed a new app called IBDsmart to help patients with ulcerative colitis or Crohn's disease receive care remotely.…
According to a story from Pulmonology Advisor, a recent study has evaluated risk factors for the appearance of bronchiectasis in patients with aspirin-exacerbated respiratory disease (AERD). A small number of…
For the last few months, we've been inundated with stories of COVID-19, and with good reason, considering the global pandemic reached over 5 million cases worldwide as of this…
Being at peace with ourselves and our lives makes daily living so much easier. Of course, sometimes it's easier said than done. But one tactic may help improve comfort, confidence,…
Passage Bio has been granted the Rare Pediatric Disease designation for their GM1 gangliosidosis treatment, PBGM01. This designation means that Passage will receive a priority review voucher, and it is…
Jalee Pelissier lives with muscular dystrophy. When she was just 13 she became an advocate for others with disorders or disabilities like her own. Now 20, her advocacy has only…
By Danielle Bradshaw from In The Cloud Copy For the past five years, it has become more and more apparent that some people have a greater predisposition to suffer from…
After six decades of attempting to solve chronic organ rejection, researchers at Houston’s Methodist Hospital and Pittsburgh University have met the challenge. According to an article in Medical Xpress,…
By Danielle Bradshaw from In The Cloud Copy Researchers in Germany have gone on record saying that there is a concern with how well patients are able to tolerate the…
By Jodee Redmond from In The Cloud Copy The results of a new study using laboratory rats to model cerebral palsy with spasticity (muscle stiffness) accompanied by dystonia (involuntary muscle…
The medical realm is changing - but how does that impact patient care and clinical expectations? The National Hemophilia Foundation discusses how clinical expectations for patients with hemophilia don't necessarily…
Genetic research is a burgeoning and exciting field. With the discovery of new genetic mutations and variants comes a deeper understanding of rare diseases and their subtypes. According to Cancer…
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