Creating a ‘bespoke’ approach to rare diseases

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Up until recently the word “bespoke” meant just one thing to me, a hand-made suit, customized and fitted to you. There’s a street in London, Saville Row, that specializes in these suits. They’re gorgeous. They’re also very expensive and so I thought I’d never have a … Continue reading Creating a ‘bespoke’ approach to rare diseases

Stem cells help researchers map out glaucoma in search for new treatments

Glaucoma is the world’s leading cause of irreversible blindness. There is no cure and current treatments are only able to slow down the progression of the disease. Now research using stem cells to create a genetic blueprint of glaucoma is giving scientist a powerful new tool to combat the disease. Glaucoma occurs when healthy retinal … Continue reading Stem cells help researchers map out glaucoma in search for new treatments

Join us to hear how stem cell and gene therapy are taking on diseases of aging

It is estimated that as many as 90 percent of people in industrialized countries who die every day, die from diseases of aging such as heart disease, stroke, and cancer. Of those still alive the numbers aren’t much more reassuring. More than 80 percent of people over the age of 65 have a chronic medical … Continue reading Join us to hear how stem cell and gene therapy are taking on diseases of aging

The long road to developing a therapy for epilepsy

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Good science takes time. That’s an important guiding phrase for researchers looking to develop new therapies. But it’s also a frustrating reality for patients who are waiting for something to help them now. That point was driven home last week when the governing board of the … Continue reading The long road to developing a therapy for epilepsy

The power of the patient advocate: how a quick visit led to an $11M grant to fund a clinical trial

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Members of NFOSD visiting UC Davis in 2013 At the California Institute for Regenerative Medicine (CIRM) we are fortunate in having enough money to fund the most promising research to be tested in a clinical trial. Those are expensive projects, often costing tens of millions of … Continue reading The power of the patient advocate: how a quick visit led to an $11M grant to fund a clinical trial

Replacement brain cells offer hope for Parkinson’s treatment

A colony of iPSCs from a Parkinson's patient (left) and dopaminergic neurons made from these iPSCs (right) to model PD. (Image credit: Jeanne Loring) A new study that used adult blood stem cells to create replacement brain nerve cells appears to help rats with Parkinson’s. In Parkinson’s, the disease attacks brain nerve cells that produce … Continue reading Replacement brain cells offer hope for Parkinson’s treatment

Smoking marijuana could be bad for your heart, but there is an unusual remedy

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Smoking medical marijuana: Photo courtesy Elsa Olofsson Millions of Americans use marijuana for medical reasons, such as reducing anxiety or helping ease the side effects of cancer therapy. Millions more turn to it for recreational reasons, saying it helps them relax. Now a new study says … Continue reading Smoking marijuana could be bad for your heart, but there is an unusual remedy

The bootcamp helping in the fight against rare diseases

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Emil Kakkis at the Rare Entrepreneur Bootcamp Imagine you or someone you love is diagnosed with a rare disease and then told, “There is no cure, there are no treatments and because it’s so rare no one is even doing any research into developing a … Continue reading The bootcamp helping in the fight against rare diseases

Turning back the clock to make old skin cells young again

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Diljeet Gill, photo courtesy Babraham Institute, Cambridge UK Sometimes when I am giving public presentations people ask if stem cells are good for the face. I always say that if stem cells could help improve people’s faces would I look like this. It’s a line … Continue reading Turning back the clock to make old skin cells young again

Recovery from muscle loss injuries hindered by immune cell conflicts

During a game in 2018, Alex Smith suffered a compound fracture that broke both the tibia and fibula in his right leg. The gruesome injury aside, the former 49ers quarterback soon developed life-threatening necrotizing fasciitis — a rare bacterial infection — that resulted in sepsis and required him to undergo 17 surgeries. In a battle to save his … Continue reading Recovery from muscle loss injuries hindered by immune cell conflicts