Updates

March 4, 2021March 4, 2021
Clinical Trials Advance as Tesomet Granted FDA’s Orphan Drug Status

Tesomet, a potential treatment for Prader-Willi syndrome (PWS), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). Saniona, the therapy’s ... Read more
March 2, 2021March 2, 2021
Mental Rigidity in PWS Goes Beyond Intellectual Disability

Adults with Prader-Willi syndrome (PWS) have greater mental rigidity than those with matching intellectual disability, but without the disease, and those with normal intellectual level, according ... Read more
February 26, 2021February 26, 2021
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a ... Read more
February 25, 2021February 25, 2021
Soleno Looking to Expand Potential Therapies That Work Like DCCR

Soleno Therapeutics has partnered with Vanderbilt University to develop therapies that work as ATP-dependent potassium channel activators for rare diseases, similar to the diazoxide choline controlled release ... Read more
February 23, 2021February 23, 2021
PWS Subtypes May Respond Differently to Psychotropic Treatments

A small study exploring gene alterations related to Prader-Willi syndrome (PWS) suggests that subtypes of the disorder have different genetic profiles that may affect response ... Read more

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October 25, 2018October 26, 2018

Lean Muscle Mass Factors Strongly in Bone Health, Study Using Prader Willi Patients as Model Finds

News

Lean muscle mass is a strong predictor of bone mineral density, or bone health, research that used Prader-Willi syndrome (PWS) as a genetic model to determine which factors influence such density reports. In fact, lean mass was found to be ... Read more

Updates

Clinical Trials Advance as Tesomet Granted FDA’s Orphan Drug Status

Mental Rigidity in PWS Goes Beyond Intellectual Disability

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Soleno Looking to Expand Potential Therapies That Work Like DCCR

PWS Subtypes May Respond Differently to Psychotropic Treatments

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Growth Hormone Therapy Also Benefits Adults, IPWSO Argues

Tonix Licenses Via-the-nose Treatment Technology From Inserm

Tryp Therapeutics Readies First Trial of TRP-8802 for Excessive Appetite

Pandemic Won’t Stop Rare Disease Day on Feb. 28

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COVID-19 Pandemic ‘Significantly’ Affected DCCR Phase 3 Trial, Soleno Says

Antidepressant Sertraline May Help Control Temper Outbursts

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

As Children Grow Up and Gain Weight, Family Life Suffers, Study Reports

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Growth Hormone Therapy Appears Safe and Helpful for PWS Adults

Diabetes Finding in PWS Patient Underscores Need for Antibody Testing

Higher Risk of Autism Spectrum Disorder Found in PWS Children

NORD’s Caregiver Respite Program Continues Through Pandemic

Art Contest Salutes Creativity of Rare Disease Communities

Online Format Outlined for PWSA’s 2021 Convention

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

INV-101 Granted FDA Rare Pediatric Disease Designation

Radius Planning Pivotal Trial of Cannabidiol RAD011

Remote Play Program May Improve Cognition, Behavior in PWS Children

EURORDIS Award Contest Welcomes Photos of Life With Rare Disease

Early, Sustained Growth Hormone Treatment Linked to Greater Benefits in Real-world Study

Mediterranean Diet, Exercise Reduce Weight, Cholesterol Levels in PWS Patients, Study Shows

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Genetic Subtypes Determine Frequency of Psychiatric Behaviors in PWS, Study Finds

PWSA USA Hosting Dec. 13 Virtual ‘Hope for the Holidays’ Benefit Concert

Lean Muscle Mass Factors Strongly in Bone Health, Study Using Prader Willi Patients as Model Finds

Clinical Trials Advance as Tesomet Granted FDA’s Orphan Drug Status

Mental Rigidity in PWS Goes Beyond Intellectual Disability

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Soleno Looking to Expand Potential Therapies That Work Like DCCR

PWS Subtypes May Respond Differently to Psychotropic Treatments

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Growth Hormone Therapy Also Benefits Adults, IPWSO Argues

Tonix Licenses Via-the-nose Treatment Technology From Inserm

Tryp Therapeutics Readies First Trial of TRP-8802 for Excessive Appetite

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Subscribe to our mailing list

COVID-19 Pandemic ‘Significantly’ Affected DCCR Phase 3 Trial, Soleno Says

Antidepressant Sertraline May Help Control Temper Outbursts

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

As Children Grow Up and Gain Weight, Family Life Suffers, Study Reports

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Growth Hormone Therapy Appears Safe and Helpful for PWS Adults

Diabetes Finding in PWS Patient Underscores Need for Antibody Testing

Higher Risk of Autism Spectrum Disorder Found in PWS Children

NORD’s Caregiver Respite Program Continues Through Pandemic

Art Contest Salutes Creativity of Rare Disease Communities

Online Format Outlined for PWSA’s 2021 Convention

Longer Use of Intranasal Oxytocin Helped Boys With PWS

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

INV-101 Granted FDA Rare Pediatric Disease Designation

Radius Planning Pivotal Trial of Cannabidiol RAD011

Remote Play Program May Improve Cognition, Behavior in PWS Children

EURORDIS Award Contest Welcomes Photos of Life With Rare Disease

Early, Sustained Growth Hormone Treatment Linked to Greater Benefits in Real-world Study

Mediterranean Diet, Exercise Reduce Weight, Cholesterol Levels in PWS Patients, Study Shows

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Genetic Subtypes Determine Frequency of Psychiatric Behaviors in PWS, Study Finds

PWSA USA Hosting Dec. 13 Virtual ‘Hope for the Holidays’ Benefit Concert