The Newborn Screening Saves Lives Act was originally introduced to facilitate the spreading of awareness and education related to newborn disease screenings. The screening of newborns has had a major…
According to a story from Scimex, the Children's Medical Research Institute in Sydney, Australia, has recently agreed to a new partnership with LogicBio Therapeutics, a biotechnology company from Boston, MA.…
According to a story from rdmag.com, the biopharmaceutical company IntraBio Inc., recently announced that its IB1000 series of investigational therapeutic compounds have been granted Orphan Medicinal Drug Designation as a…
Eltrombopag (Promacta) has now been approved for all individuals in the US with severe aplastic anemia. The drug will be accessible to both pediatric and adult patients. This is huge news.…
According to a story from CheckOrphan, a wide array of experts are raising concerns about India's rare disease policy, which was first unveiled in May of 2017. Despite being hailed…
Glioma is a type of cancerous tumor that originates in the brain. It has various types and the tumors range in aggressiveness. There are around 11,000 people just in the…
We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…
According to a story from The Oncology Times, the US Food and Drug Administration (FDA) has been moving forward with several treatments and potential treatments for a variety of different…
Limb-Girdle Muscular Dystrophy or LGMD, is a form of Muscular Dystrophy caused by a mutation which disrupts production of the protein beta-sarcoglycan. There are currently no treatment options for LGMD and…
Polycythemia Vera or PV, is a rare blood cancer. It's caused by a mutation which results in the overproduction of red blood cells. Too many blood cells significantly increases your…
Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…
According to a story from Xconomy, the Rainwater Foundation, which was first founded in the 90s by Texas billionaire Richard Rainwater, is offering cash rewards to the tune of $250,000…
REGENXBIO is a biotechnology company that has been developing a technology platform for gene therapy called NAV. AAV9 is one of the vectors being tested with NAV technology as a…
Batten disease is a lysosomal storage disorder. It's neurodegenerative, meaning it results in the deterioration of neurons. One of the most common forms of this disease is caused by mutations in the…
According to a story from the Australian Jewish News, a Jewish teen named Gidon Goodman was born with Gaucher disease. Recently, he started the first Gaucher disease patient support group…
According to a story from Benzinga, the drug developers AstraZeneca and Merck have recently announced that the US Food and Drug Administration (FDA) has decided to accept a supplemental New…
According to a story from BioSpace, the gene therapy company Rocket Pharma recently announced that its investigational product candidate RP-L102 recently saw its Investigational New Drug (IND) application gain approval…
Often times it seems that research for the rarest of conditions is brushed aside. Primary attention is given to more common illnesses which affect more people. For instance, in the…
According to a story from BioSpace, the biotechnology company Partner Therapeutics, Inc., recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to the company's…
According to a story from Hematology Times, an experimental CAR-T cell therapy called P-BCMA-101 was recently granted regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration.…
According to a story from prweb.com, the Neuromuscular Disease Foundation (NDF) recently announced that is has earned a $2.5 million challenge grant. This massive grant will go towards funding the…
BioMarin has just released an update on their achondroplasia and hemophilia clinical trials. Exciting things are in the works! Achondroplasia Update Achondroplasia causes short stature. A phase 3 trial working to…
In 1993, researchers found one of the causes of familial amyotrophic lateral sclerosis (ALS). ALS is a neurodegenerative disorder which affects the motor neurons. Motor neurons are responsible for transmitting…
Happy Friday! Winter is coming and so is FDA news! Take a moment to check out some recent FDA announcements. FDA Approves Triplet Immunotherapy Combination for Multiple Myeloma Big news…
According to a story from Trend News World, mother Orvelina Lizeth Melendez is heading north as part of the migrant caravan that has been making headlines as it makes its way…
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