Editor’s Choice: Rare Disease Events and Finding Growth Through Adversity
The Dog Days Are Back Is it hot or what!? Whether you're trying to catch a tan or stay cool, we have a fresh dose of weekly content in Editor's…
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Experimental Netherton Syndrome Treatment Receives Rare Pediatric Disease Designation from FDA
According to a press release from LifeMax Laboratories, Inc., the Food and Drug Administration (FDA) has granted the Company's experimental Netherton syndrome drug LM-030 (licensed from Novartis) Rare Pediatric Disease…
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Antibodies: The Key to Fighting Sickle Cell Anemia? Approval Could be Just Months Away
According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This…
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Plan proposed to use CRISPR to edit gene carrying HIV by Russian Biologist
As reported in RadioFreeEurope, Russian biologist Denis Rebrikov is currently challenging the norm of caution regarding gene-editing in the science world, with efforts to gain the go-ahead from Russia…
Dealing with Needle Anxiety? This Device Might Help
If you have ever spent time sitting in your doctor’s office with a nurse hovering over you struggling to find a vein in your arm to take blood, you can…
This Couple Has Been Advocating for Primary Immunodeficiency Disease Since the 90’s
Vicki and Fred Modell have spent years since their son’s death in the 90's advocating for more research and assistance to be provided to those with primary immunodeficiency disease. Their…
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New “Wrist Watch” Will Monitor Parkinson’s Disease and Connect Patients to Doctors Remotely
Parkinson’s disease can be a hard disease to manage. Not only does it take a physical and emotional toll on patients and loved ones, but managing every day activities can…
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Promising Drug for Sickle Cell Disease Heads Towards Approval
As recently reported in CheckOrphan, a new drug for sickle cell disease has quickly ascended through the FDA approval processes in an accelerated review. A New Sickle Cell Drug Shroud…
Watch: 6-Year-Old Has Disease So Rare, Doctors Named it After Him
Meet 6-year-old Grayson Kole Smith -- who after 36 surgeries and a grim prognosis has overcome the odds to live his life. Grayson was born with several serious medical issues…
Patients with Amyotrophic Lateral Sclerosis Can Now Change the TV Channel with Just Their Eyes
For rare disease patients who face physical disabilities, even seemingly simple tasks can pose extreme difficulties. For instance, changing the channel on the television. Comcast Corp. previously created a voice…
NORD 2019 Living Stronger Forum Highlights
The 2019 NORD Living Stronger Forum was held in Houston, TX, at the end of June. The event brought families and physicians from all over for educational presentations, community, and…
Catalysts for Change and Guidance for Growth: Terri’s Story
Terri considers herself “very lucky”. In the 80s her paternal grandmother and her grandmother’s two sisters were diagnosed with ovarian cancer. In the 90s, her family entered into the BRCA1…
Celebrate Gastroschisis Awareness Month with Avery’s Angels Foundation
July is Gastroschisis Awareness Month! Thanks to the great work of the Avery's Angels Gastroschisis Foundation, there are a number of ways and resources to make the most out of…
New Rare Disease Discovery, Made Possible By Research
The San Francisco Chronicle had a pretty neat story about how one man's post-cancer neurological issues lead to a so-rare-it's-still-unnamed disease diagnosis. And tracking how we got to the movement…
Florida State Football Team Hosts Fundraising Event for Rare Disease Research
Any Noles fans? Well, prepare to love them even more. Last weekend, players on the Florida Statue University Seminoles football team participated in FSU’s annual “Lift for Life” event, which raises…
Maine Camp Hosts Families with Rare Diamond-Blackfan Anemia
Some of us here may have heard of Camp Sunshine -- which provides retreats "combining respite, recreation and support, while enabling hope and promoting joy, for children with life-threatening illnesses…
Genetic Data Collection for Rare Diseases Gets Multi-Million Dollar Grant
The NIH has awarded Geisinger Health System, along with the University of Washington in Seattle and Washington University in St. Louis, a $5.8 million, five-year grant to study the role of…
Study: Rare Disease Drug Development Outpacing Approval/Execution, Changes Needed
Rare disease drug development, which now accounts for nearly one-third of all drugs in active research & development worldwide, presents challenges that will accelerate the adoption of new development strategies…
The Rare Barometer Program Helps Politicians Stay Informed on Issues Important to Rare Disease Patients
Eurordis-Rare Diseases Europe Eurordis-Rare Diseases Europe is an alliance of patient organizations which works to give rare patients a voice. It also strives to spread awareness of rare diseases to…
University of Michigan Professor Creates Website to Provide Scleroderma Patients Resources and Support
Dinesh Khanna, a professor at the University of Michigan has worked heavily with the rare disease scleroderma. Michigan has its own Scleroderma Program, of which Khanna is the director. Through…
Editor’s Choice: So What’s the Deal with Kratom?
Happy 7-11 Day! We hope everyone's on their way to get their free slurpie today! For this week's editor's choice, we're highlighting four articles. First, we discuss kratom, the controversial…
Multiple Myeloma Drug ‘Xpovio’ Passes With FDA Accelerated Approval
As reported in Biospace, xpovio (selinexor), a drug used to treat relapsed refractory multiple myeloma, received accelerated approval by the Food and Drug Administration this July. About Multiple Myeloma Multiple…
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Herbal Supplement ‘Kratom’ Used as Pain-Relieving Alternative, Receives Further Research Funding
Kratom, an herbal supplement of the caffeine family used to ward off pain for chronic pain conditions, has received large grants for further research. While many consumers claim the supplement…
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Recent Study Finds Better Than Expected Safety Data for TNF Inhibitors in Ankylosing Spondylitis
According to a story from Ankylosing Spondylitis News, a recent study has found that the class of drugs known as tumor necrosis factor (TNF) inhibitors may actually be safer for…
FREE MDS Forum in Iowa City!
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. The event will take place in Iowa City, Iowa on Saturday July 20th, 2019. Event Summary:…
