Veer Basal, age 14, first started showing signs of Friedreich's ataxia when he was around ten years old. The first sign that something was wrong was when Veer began to…
In a recent press release, biopharmaceutical company ASLAN Pharmaceuticals ("ASLAN") announced that it would begin recruiting patients for an expansion cohort of a clinical trial evaluating ASLAN004 for patients…
According to a recent article in Statnews, providers must obtain prior authorization from an insurer for several reasons; to start, the insurer often requires physicians to justify prescribing a…
Turkish citizens are putting pressure on their government in regards to access to rare disease treatments, specifically spinal muscular atrophy (SMA) therapies. There are currently two drugs available to Turkish…
According to a story from dernieresactus.fr, the biopharmaceutical company Inventiva has recently announced plans for its upcoming phase III clinical trial. This trial will investigate its lead candidate lanifibranor as…
Recently, clinical stage biotechnology company Prilenia Therapeutics B.V. ("Prilenia") announced that the first patient has been enrolled in a Phase 2/3 clinical trial evaluating pridopidine for patients with amyotrophic…
In some cases, combining different therapies can provide better overall outcomes for patients. However, in a Phase 2 clinical trial evaluating bevacizumab in conjunction with osimertinib for patients with EGFR-mutated…
One key struggle with managing rare diseases or other health conditions is finding the right treatment option. While bronchiectasis is usually treated with antibiotics and chest physiotherapy, there seems…
According to BioSpace, recent research has demonstrated that CRISPR gene editing could be the answer to treating progeria, or Hutchinson-Gilford progeria syndrome. The Broad Institute and NIH have collaborated to…
While novel treatments have been rapidly emerging onto the drug scene, new treatment options for pancreatic cancer have fallen behind. But now, according to OncLive’s interview with Dr. P.A. Philip,…
Parents of children who have received a diagnosis of Hutchinson-Gilford progeria syndrome (HGPS) may be relieved to learn the FDA recently approved Zokinvy capsules that were developed to lower…
The initial CRISPR treatment for sickle cell disease was administered to Victoria Gray in July 2019. Victoria, a resident of Forest, Mississippi, was born with sickle cell and has…
According to a story from Charcot-Marie-Tooth News, a recent cell-based study has revealed that Charcot-Marie-Tooth disease type 1C and type 4J share a common pathway malfunction. This is despite the…
The FDA has recently cleared an Investigational New Drug (IND) application for FBX-101, allowing it to move into a Phase 1/2 clinical trial. This gene therapy was developed for the…
12-year-old Aidan Carter has Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), a rare, progressive condition that impacts mental development, appearance, physical abilities, and organ function. In order…
A recent announcement by Gilead Company citing data from Kite Pharma, one of its premier companies, set out remarkable results from Kite’s Zuma-1 clinical trial. The findings confirmed that…
As reported in PR NewsWire; in an effort to help the entirety of the sickle cell disease community, Emmaus Life Sciences, Inc has announced a program that will provide their…
The Neurological Alliance has released a report that details the experiences that rare, neurological disease patients in the UK share and calls for improvements for these people. Experts in the…
Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is a rare disease that causes progressive muscle loss and resultant muscle weakness. It is caused by a lack of motor neurons. It…
According to a story from Targeted Oncology, Dr. Bradley Monk talked about the importance of molecular testing in determining the treatment course for ovarian cancer at the Targeted Oncology Case…
Landos Biopharma has recently released data from their Phase 2 trial of BT-11, a treatment for ulcerative colitis. The results were positive, making this therapy a possible viable treatment for…
During the drug development process, drug manufacturers must seek out an Investigational New Drug application (IND), which allows the treatment to be transported across state lines. IND clearance also…
According to a recent report in Cancer Network, the results from CARTITUDE-1 were well received. CARTITUDE-1 is a phase 1b/2 clinical trial of the CAR T-cell treatment, Cilta-Cel, for the…
According to GlobeNewswire, Health Canada has approved of a biosimilar, Hyrimoz, to treat ankylosing spondylitis and eight other rare conditions. Sandoz Canada is excited about this approval, as it expands…
Many rare disease patients understand the financial burdens of living with a rare disease. As the therapies are only supporting a small number of patients, the price tag is often…
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