Global Blood Therapeutics, Inc. (GBT) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to voxelotor (previously called GBT440) for the treatment of sickle cell disease (SCD). Voxelotor is being developed as a disease-modifying therapy for SCD and previously received European Medicines Agency (EMA) Priority Medicines (PRIME) designation for the treatment of SCD. “The FDA’s decision to grant voxelotor the first Breakthrough Therapy designation for the treatment of sickle cell disease reflects a recognition of the promising efficacy and safety data we have collected to date for this investigational drug, as well as an acknowledgement of the overwhelming need for major advances over available therapies in the treatment of SCD patients,” said Ted W. Love, president and chief executive officer of GBT. “This designation is another significant milestone for GBT as we work to expedite the development of voxelotor.”

Emmaus Life Sciences has announced that Endari, a medication developed to reduce acute complications in patients with sickle cell disease, is now available by prescription in the United States.

Sickle cell disease is an inherited blood disorder in which the body produces crescent shaped red blood cells that get caught in blood vessels, ultimately blocking blood flow. An altered form of hemoglobin caused by genetic mutations makes red blood cells fibrous and rigid which forms their abnormal shape.

Sickled red blood cells do not last as long as normal red blood cells, causing anemia to develop in patients. Signs that a patient could have sickle cell disease begin in early childhood and include anemia, repeated infections, and periodic episodes of pain, clinically defined as sickle cell crises.

 

A man and woman who are both carriers of the sickle cell anemia mutation can have a healthy child by fertilizing eggs in a lab, then deciding which of multiple embryos that develop from the eggs will become the baby, a case study indicates. It also showed that umbilical cord blood stem cells from a healthy baby born this way could be used to treat a brother or sister with sickle cell anemia. Sickle cell anemia is an autosomal recessive disease, meaning that a child has to inherit two mutated copies of the hemoglobin gene to develop it — one from each parent. If a child inherits one mutated copy and one normal copy, they will have sickle cell trait, a condition that generally shows no symptoms.

In efforts to assist in spreading holiday cheer, SCDAI hosts a Toy Drive that receives donations from businesses both large and small, as well as private and corporate organizations.

To culminate the event, for the past three decades SCDAI has held a Holiday Celebration for individuals with Sickle Cell Disease. This celebration is arranged so that individuals with sickle cell disease and their families can share with SCDAI in the joy of the holidays.

 

Date:
Sunday, December 19, 2021

 

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Twin brothers and New England Patriots stars, Devin and Jason McCourty are hosting the 2019 Tackle Sickle Cell Casino Night in Boston on Monday, October 28, 2019. This fundraiser takes place from 7:00 p.m. – 10:00 p.m. (VIP Red Carpet at 6:30pm) at Royale, located at 279 Tremont Street in Boston and benefits two Boston area nonprofits, Boston Medical Center and Next Step.

The McCourty Twins Tackle Sickle Cell campaign aims to educate the public and increase blood donors, as well as raise money and awareness for the fight against sickle cell disease.

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Now in its 12th year, Rare Disease Week on Capitol Hill empowers and inspires hundreds of advocates each year. The connections you make during the week will impact rare disease patients for generations to come.

Hosted by the Rare Disease Legislative Advocates (a program of the EveryLife Foundation for Rare Diseases), the multi-day event brings together rare disease advocates from across the country to make their voices heard by their Members of Congress. Participants are educated on policy proposals impacting the rare disease community and provided opportunities to advocate for policy changes directly to their Members of Congress. No matter one’s connection to rare disease or their advocacy experience level, all are welcome.

Date:
FEBRUARY 28TH – MARCH 2ND, 2023

 

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Rare Disease Day takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives.

The campaign targets primarily the general public and also seeks to raise awareness amongst policy makers, public authorities, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases.

Rare Disease Day events are down to hundreds of patient organisations all over the world who work on a local and national level to raise awareness for the rare disease community in their countries.

 

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2021 Fall program schedule to be determined. Session dates may be subject to change at Camp Sunshine’s discretion. We encourage you to visit this site periodically or contact us directly, as there may updates or additions to the schedule over the course of the year.

Camp Sunshine at Home: Sickle Cell Disease - August 16, 2021 to August 19, 2021

 

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The study looked at Seidman's BEATS (Build, Educate, Advance, Transition in Sickle cell disease) music therapy program, developed by Rodgers-Melnick. BEATS focuses on young adults ages 18-35 who have transitioned from pediatric care at Rainbow Babies and Children's Hospital to an adult care program at Seidman.

To help young adults with their transition to adult care, doctors at Seidman developed the "Bridge Clinic," a care team that includes a hematologist, nurses and social worker, as well as one-hour BEATS music therapy sessions that Rodgers-Melnick leads.

Each week, BEATS participants create a different song about how the disease affects the body, what to do if they develop a fever or pneumonia, pain management techniques and other topics. The program currently has 50 participants.

The Sickle Cell Disease Association of America, Inc. (SCDAA) is proud to announce Pennsylvania native Kiarra Roseburgh as SCDAA’s National Child Ambassador.

The SCDAA poster child tradition started in 1976 as an opportunity to elect goodwill ambassadors who would serve as emissaries and assist in public education campaigns. A nationwide search takes place biennially to find the next child who will give voice to millions of children who suffer from sickle cell disease (SCD).