New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…
According to a recent article featured in Biospace, researchers found substantial evidence of a biochemical correction after treating an eleven-year girl for Gaucher Disease. This is the first pediatric case…
Parents and doctors were concerned about Chuck Goodman's health from an early age. In fact, they thought he had a deadly case of childhood leukemia. At around age five, Chuck…
The FDA created the Rare Pediatric Disease Designation and Priority Review Voucher Program to stimulate the development of therapies intended to treat, diagnose, or prevent rare pediatric diseases. These are…
The joy of being told you have a normal healthy newborn can at times be overshadowed by concern about whether the baby has a yet-to-be-discovered disease. Medical technology has helped…
According to a story from Kalimpong News, over 300 children with rare diseases in India are still waiting for the release of pledged funds from the Union Health Ministry so…
As we know, many treatments for rare diseases are quite expensive - and this price doesn't even factor in travel, lodging, appointments, and more. To help with this, the government…
Compiling data, especially rare disease data, can be extremely helpful for research purposes and for doctors treating patients with that condition. Recently, the International Gaucher Alliance and Cerner Enviza have teamed…
One of the burdens that rare disease patients often face falls into the financial category, and that's treatments that come at a very high cost. One patient, a young girl…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
From November 21st until the 23rd, medical professionals and others involved in the metabolic field will come together for the 14th International Congress of Inborn Errors of Metabolism (ICIEM). It…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare or life-threatening illnesses, defined as those affecting under 200,000 people or…
In a recent news release, rare disease-focused biopharmaceutical company CANbridge Pharmaceuticals Inc. ("CANbridge") shared that the National Medical Products Administration (NMPA) of China accepted an Investigational New Drug (IND) application…
In mid-April, Kantar Health and the International Gaucher Alliance (IGA) shared the launch of a rare disease registry developed by the partnership. The registry, known as GARDIAN, will explore Gaucher…
At the American Society of Hematology Annual Meeting in 2020 (ASH 2020), the latest info and data related to Gaucher disease, a form of lysosomal storage disease, was presented. It…
Prevail Therapeutics, a company that focuses on developing gene therapies for neurodegenerative diseases, has announced that a composition of matter patent has been issued for PR001. This therapy is intended…
According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…
According to a story from Parkinson's News Today, a recent study may have discovered the origins of chronic pain and damage to the sensory nerves as found in some cases…
Gaucher Disease Awareness Month takes place every October, but amidst this new global pandemic, planning has taken place early to figure out how to make the month as successful as…
CytRx Corporation has released the results of the second phase of their study of arimoclomol as a treatment for Gaucher disease. The data was positive, and it demonstrated this therapy's…
According to a story from globenewswire.com, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the…
CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis…
Have you been feeling a little stiff or achy later? Maybe tired? I know that my body has undertaken a lot of stress thanks to the new life changes -…
Rare, neurologic diseases often do not have viable treatments. Gaucher disease and Parkinson's disease are two examples. In situations like these, medical professionals look to treatments for other conditions, such…
Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…
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