Parkinson’s Disease Phase 2 Clinical Trial Currently Enrolling
A new Phase 2 Parkinson's disease trial is now enrolling 42 patients who have lived with the condition four years or more. Ultimately, the trial aims to evaluate a new…
CZI has Committed 64 Million Dollars to the Research of Neurodegenerative Disorders
Neurodegenerative disorders affect millions of people across the world. They include Amyotrophic Lateral Sclerosis, Parkinson's disease, Huntington's disease, and Alzheimer's disease, among others. For most neurodegenerative diagnoses, there are no effective treatments, no…
Research Indicates Sex Differences Affect Response to Cancer Treatment
For a long time, we've known that, at least in North America, men develop cancer at higher rates than women. However, there is still some mystery concerning the impact of…
Angelman Syndrome Clinic Network is Now International
Angelman Syndrome (AS) is a neurogenetic disorder. It's not fatal, but prevents patients' from living independently. It causes neurological impairment, seizures, and balance issues among other problems. It's caused by a…
Charcot-Marie-Tooth: Misconceptions from People Who Just Don’t Know
Charcot-Marie-Tooth disease (CMT), is a rare neurological condition. It's hereditary, and it affects the way nerves communicate with muscles. There are different types of CMT, determined by which genes are…
Phase 3 Clinical Trial for Angelman Syndrome Coming in 2019!
Angelman syndrome impedes the brain's ability to distinguish background noise from whats important. This is a result of a reduced tonic inhibition, caused by a loss of function of the…
Cosmetology Students Triple Supply of Wigs for Cancer Patients
In August, Duke Cancer Center's Patient Support Program had a backlog of 150 wigs. That means 150 cancer patients who didn't have access to an item that, while simple, provides…
Hereditary Angioedema Discovery is First Step Toward Gene Therapy
Hereditary Angioedemia (HAE) results when the blood is missing the C1-inhibitor protein. It causes unpredictable and sudden attacks of swelling on various parts of the body. It can affect practically…
The Importance of a Patient Registry for Lyme Disease: MyLymeData
To be quite frank, Lyme disease is an under-researched condition. If you look at the data concerning how many clinical trials are developed each year for rare conditions, Lyme disease…
New Phase 1b/2a Trial for Pulmonary Sarcoidosis is on its Way!
Sarcoidosis is an inflammatory condition which can affect any organ in people of any age. The prognosis ranges from benign to debilitating. Granulomas, or clumps of cells, build up in…
Research Updates for Multiple Myeloma and WM!
Dana-Farber Cancer Institute has just announced research updates on multiple myeloma and waldenstrom macroglobulinemia which indicate significant progress in the search for treatments for these diseases. The Findings 1) There…
Homocystinuria is More Common Than We Thought, It’s Just Not Detected Well in NBS
Most patients diagnosed with Homocystinuria (HCU), a rare metabolic disorder, don't receive their diagnosis until later in life. Scientists were at a loss for why patients with phenylketonuria (PKU), a similar…
“Just Like You” Dolls for Conditions like Cerebral Palsy
Just Like You Dolls Just Like You Dolls is a family owned business based in Australia. They design dolls to match their child owner as closely as possible. This includes…
The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy
This past Sunday, December 2nd, during the Patriot's game against the Minnesota Vikings, 20 players wore customized cleats to spread awareness for various philanthropic causes. This is the third year…
Madeline’s Spinal Muscular Dystrophy Story: A Wheelchair Can’t Slow Her Down
"This is what I have to work with. I’m going to make the best out of it.” That's Madeline Schott's motto. The 18 year old was diagnosed with Spinal Muscular Atrophy…
Pawn Shop Owner has Donated 580 Motorized Wheelchairs to People in Need
Phil Pavone owns a pawn shop in Norwich, Connecticut. It's called AZ Pawn. Now, in addition to being a businessman, he's practically created his own nonprofit. It all started one…
We Need More Racially Diverse Clinical Trials. Now.
It was in 1993, just 25 years ago, that congress passed an act to require more diversity in clinical trials. Specifically, it required more female and ethnic minority representation. Since…
Positive Results from Safety Trial of CBD for Refractory Pediatric Epilepsy
INSYS Therapeutics has just released data from their long-term safety study of CBD as a treatment for refractory pediatric epilepsy. The results were positive and we're excited about the future…
International Experts Met with Goal to Improve Care for Chronic Myelomonocytic Leukemia Patients
Chronic myelomonocytic leukemia, or CMML, is a rare condition. However, it has similarities to myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN). That being said, it still a unique diagnosis, and…
Potential New Treatment for Huntington’s Disease Granted Orphan Drug Designation
Huntington's Disease is a neurodegenerative condition caused by a genetic mutation. It results in progressive decline of motor skills. It also affects cognitive function. Patients experience decline for 15-20 years…
Positive Results from Part A of a Phase 3 Trial for Sickle Cell Disease
Sickle cell disease (SCD) is a blood disorder. SCD patients have abnormal hemoglobin, or sickle hemoglobin, caused by a genetic mutation in the beta-chain. It results in deformed and inflexible…
Rare Disease Drug Costs Evaluated by Organization’s Interpretation of Your Quality of Life
In the UK, a think tank determines access to rare disease drugs based on their perception of an individual's quality of life. That idea has also been developed in the…
Potential New Treatment for Amyotrophic Lateral Sclerosis which Doesn’t Require Swallowing!
Amyotrophic lateral sclerosis (ALS) is a condition which affects the bodies nerve cells. It's neurodegenerative and progressive, causing the patient muscle weakness and muscle atrophy. It eventually results in paralysis.…
A New Combination of Therapies has Potential to Treat all Mantle Cell Lymphoma Patients
Mantle cell lymphoma is caused by a genetic mutation which produces cancerous white blood cells. Of course, these are the cells which fight infection in a normal body. Therefore in…
Vote to Help Family Win 50,000 Dollars for Mitochondrial Disease Research!
There are only 11 people in the world diagnosed with Mitochondrial Complex 1 Deficiency. Katherine Faughn, a 7-year-old living in Kentucky is one of these 11. The disease wasn't even…
