Neurofibromatosis Type 1
NF1 Patients Still Need Proper Treatments
By Danielle Bradshaw from In The Cloud Copy Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (pNFs) is a condition that many children struggle to manage due
Rare Patient News. Well Done.
Rare Disease Spotlight: Activated PI3K Delta Syndrome
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ICYMI: Infant Death in Trial Unrelated to Spinal Muscular Atrophy Gene Therapy
ICYMI: Privigen Granted Orphan Drug Designation For Treatment of Systemic Scleroderma
Clinical Trial for Tourette Syndrome Does Not Meet Endpoints
LATEST PATIENT WORTHY ARTICLES
Rare Disease
Priority Review Granted for Relapsed and Refractory Follicular Lymphoma Treatment
According to a press release from Epizyme, Priority Review has been granted for tazemetostat (Tazverik), a drug used to treat relapsed or refractory follicular lymphoma
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Rare Disease
The EMA Grants PRIME Designation to Viralym-M To Treat Viral Infections Linked to Stem Cell Transplant
According to a recent article in The Pharma Letter, the European Medicines Agency (EMA) granted PRIME (PRIority MEdicines) designation to Viralym-M based on positive results
Alzheimer's disease without Neurofibrillary tangles
Toxic Protein Identified: Closing in on Alzheimer’s and Neurodegenerative Diseases
For years scientists have tried to understand how and why tau, well known to researchers for its implication in Alzheimer’s disease, changes from its
Rare Disease
Specific Autoantibodies Linked to Scleroderma, Research Study Finds
By Jodee Redmond from In The Cloud Copy A recent study suggests that certain antibodies could be linked to specific human leukocyte antigen (HLA) genes
Friedreich's ataxia
Key to Possibly Reversing Friedreich’s Ataxia Found
By Danielle Bradshaw from In The Cloud Copy A Tufts University science team was able to identify a means to possibly reverse Friedreich’s ataxia.
