Khondrion has just announced that the very first patient has been dosed in their Phase 2b study for mitochondrial diseases called KHENERGYZE. This patient population has a high unmet need…
The Benefits of Genomics The use of genomics in health systems is becoming more of a standard practice. Health systems have been partnering with genomics databases to collect patient data.…
FightMND is an Australian nonprofit organization dedicated to improving the lives of motor neurone disease patients through funding research for cures. They've successfully provided millions toward research initiatives and clinical…
International Alagille Awareness Day The very first International Alagille Awareness day was held on January 24th, 2020. What is Alagille syndrome? It is a rare disease which effects the heart,…
India first drafted a national rare disease policy back in 2017. Unfortunately, it was never enacted due to budgeting and implementation issues. This draft included suggestions such as a corpus…
The Platform Notable's automated technology platform was created to help predict which patients would respond better to which therapies. As no patient is the same, this study is paramount. Notable…
More than 20 years ago researchers at Harvard University, led by professor Oliver Pourquié, discovered how the vertebra in chickens first form. Essentially, a 'tick' initiates the formation of a…
Epithelioid sarcoma is a rare form (accounts for <1%) of soft tissue sarcoma. Until now, there have been no treatments specifically for this patient population. But the FDA has just…
SELLAS has just announced that patient screening has begun for a Phase 3 trial for acute myeloid leukemia (AML). The trial is examining the effects of GPS for patients who…
A New Phase 3 Trial A new phase 3 study will begin soon this year at Stanford University. Abeona Therapeutics had been waiting on IRB approval to begin the trial,…
Body Image In today's world, negative body image is unfortunately very common. Research has shown that this trend occurs in women and men, both as children and adults. It affects…
The Study: MRx0518-I-003 MRx0518-I-003 is a trial investigating MRx0518 as part of a combination treatment for pancreatic cancer, used with preoperative radiotherapy. The trial will evaluate the therapies safety and…
Sickle Cell Disease Sickle cell disease is a rare blood disorder. It is progressive, debilitating, and sadly can be life-threatening. The genetic defect leads patients' red blood cells to be…
Galactosemia Galactosemia is a rare disease which affects the metabolic system. It causes the body to not be able to process galactose properly. Galactose is a simple sugar present in…
Olmsted syndrome is a rare disease most frequently caused by mutations in TRPV3. For some patients, it is caused by MBTPS2 mutations. It is characterized by palmoplantar keratoderma (PPK) as…
The 2019 European Academy of Dermatology and Venereology Congress was held in Madrid, Spain. During this conference, a symposium was held discussing IL-23 inhibition in psoriasis. Here are some of…
In the age of booming media, it can be difficult to know or be confident that you are receiving correct information. When it comes to your healthcare or the care…
Schizophrenia patients in the United States now have a new treatment option. Intra-Cellular Therapies has just announced that their investigative therapy called CAPLYTA has received FDA approval for adult schizophrenia…
Doctor Daniel Kastner, director of NHGRI, recently uncovered a new rare disease along with his research team. It’s called CRIA syndrome. This discovery has been published in Nature. CRIA CRIA…
A New Working Group A team of healthcare professionals from the Institute for Quality and Efficacy in Health Care, the Drug Commission of the German Medical Association, and the Charité…
TrialJectory is an organization working to match bladder, breast, colorectal, melanoma, myelodysplastic syndromes (MDS), and other cancer patients with the clinical trial that is right for them. They have successfully raised…
The FDA has just provided Orphan Drug Designation to Hizentra for the treatment of adults diagnosed with chronic inflammatory demyelinating polyneuropathy (CIPD). This is a maintenance therapy which works to…
Marinus Pharmaceuticals has recently announced updates on its ganaxolone programs for tuberous sclerosis complex (TSC), PCDH19-related epilepsy (PCDH19-RE), and CDKL5 deficiency disorder (CDD). Ganaxolone Ganaxolone is a positive allosteric modulator…
David Fajgenbaum was diagnosed with Castleman disease while he was still a medical student. He was perfectly healthy, but in a few days he had organs failing. He was treated…
The EMA has just announced that Danicopan has been accepted into its PRIority MEdicines (PRIME) program. Danicopan is being developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. PRIME…
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